Includes: 
- All SageMaker Studio Features
Enhancements: 
- No-Code/Low-Code Interface
- Visualization Options

Software and apps

Creative (multimedia and design)

Ideas

Technology demonstration and hardware

Nominations

Business plans

Analytics, visualizations, algorithms

Scientific

1st Pediatric Clinic of the University of Athens

23ANDME, INC.

3Helix, Inc

8LDesign

ACADEMISCH MEDISCH CENTRUM

Acadia Clinical Research

Aclipse Therapeutics

Addenbrooke's Charitable Trust

Advanced Research and Diagnostics Laboratory

Advances in Health Inc.

AdventHealth Orlando

Ahvaz Jundishapur University of Medical Sciences

Ajou University

Akhtar Saeed Medical and Dental College

AKRON CHILDREN'S HOSPITAL

Akron Nephrology Associates

Aland Central Hospital

Albany Research Institute

ALBANY STRATTON VA MEDICAL CENTER

Alberta Children's Hospital

ALBERT EINSTEIN COLLEGE OF MEDICINE

Alder Hey Children's Hospital

ALERE INTERNATIONAL

Aligarh Muslim University

AliveDx

Allegheny Health Network

Alnylam Pharmaceuticals

Alpine Institute for Longevity Research and Diagnostics

Altru Health System

American Registry of Pathology

American University of Beirut

Am jain college

ANNA UNIVERSITY

ANN & ROBERT H. LURIE CHILDREN'S HOSPITAL

Ann & Robert H. Lurie Children's Hospital of Chicago

Arab Academy for Science, Technology and Maritime Transport

Arbor Research Collaborative for Health

ARCHIMEDES, INC.

ARIZONA STATE UNIVERSITY

Arkansas Children's Hospital

Arnold Palmer Hospital for Children

Arnold Palmer Pediatrics Endo

Asheville Gastroenterology Associates

ASSOSTANCE PUBLIQUE HOPITAUX DE PARIS

Astral Diagnostics

ASTRAZENECA

Atlanta Diabetes Association

Atlanta VA Medical Center

Auckland Diabetes Centre

Audie L. Murphy Memorial VA Hospital

Aukland Diabetes Centre

AURA Paris

Autonomous University of Zacatecas

BAKER HEART AND DIABETES INSTITUTE

Ballad Health

Ballet Company

Baltimore VA Medical Center

Banner Health

Banner - University Medical Center Phoenix

Barbara Davis Center for Childhood Diabetes

Basingstoke and North Hampshire Hospital

BAXTER HEALTHCARE CORPORATION

BAYLOR COLLEGE OF MEDICINE

Baylor University Medical Center

Baystate Medical Center

BC Children's Hospital Research Institute

BECKMAN COULTER, INC.

BENAROYA RESEARCH INSTITUTE

Bender Tech, LLC

Bend Memorial Clinic

Benedictine College

BEN GURION UNIVERSITY 

BETH ISRAEL DEACONESS MEDICAL CENTER

BG University Hospital Bergmannsheil Bochum

Billings Clinic Ped. Diabetes Ctr.

BioAgilytix

Biomedical Engineering Department, Medical Research Institute, Alexandria University

Birmingham Children's Hospital

B.N.M Institute of Technology

Booth Hall Children's Hospital

Booz Allen Hamilton

BOSTON CHILDREN'S HOSPITAL

BOSTON MEDICAL CENTER

Boston Scientific

BOSTON UNIVERSITY

Bowman Gray School of Medicine

Brandeis University

Breakthrough T1D

BRESMED

BRIGHAM & WOMEN'S HOSPITAL

Brighton and Sussex Medical School

BRISTOL UNIVERSITY (UK)

BROAD INSTITUTE

BROWN UNIVERSITY

BU-ALI SINA UNIVERSITY

BUTLER UNIVERSITY

CAIRO UNIVERSITY

Çanakkale Onsekiz Mart Üniversitesi

Canberra Hospital

CAPISH INC

PraktischGut

NIH Test

NIDDK

National Institute on Aging

NIAMS

NICHD

NIDCD

NIH CLINICAL CENTER

National Institutes of Health (NIH) 

National Institute of Allergy and Infectious Diseases (NIAID)

National Institute of Environmental Health Sciences (NIEHS)

Indian Institute of Technology Mandi

National Center for Biotechnology Information

National Institute on Alcohol Abuse and Alcoholism

NIDDK - Phoenix Epidemiology & Clinical Research Branch

Cash Prize

Non-monetary

Both cash prize & non-monetary

The African American Study of Kidney Disease and Hypertension study (AASK Trial) was a multi-center, randomized clinical trial that investigated the effects of blood pressure (BP) control and the use of specific antihypertensive regimens on the progression of chronic kidney disease (CKD).  The study was designed to address the high incidence of CKD in African Americans with hypertension. A two by three factorial study design, including two levels of BP control as defined by mean arterial pressure (MAP) and three different anti-hypertensive regimens, was used. Participants were randomized into two levels of BP control groups, either a goal MAP < 92 mmHg or a goal MAP between 102-107 mmHg inclusive.. The antihypertensive drugs used for treatment were amlodipine, a calcium channel blocker; ramipril, an angiotensin-converting enzyme inhibitor; and metoprolol, a β-blocker. The trial was double-blind (drug administration only). The change in glomerular filtration rate (GFR) as determined by iothalamate clearance was measured as the primary outcome to test the regimens’ efficacy in slowing the rate of decline of kidney function.
 
The study showed that the angiotensin-converting enzyme inhibitor ramipril was more effective than the beta blocker metoprolol or the dihydropyridine calcium channel blocker amlodipine in slowing the rate of GFR decline. The results suggest that reduction in blood pressure to levels below those currently advocated for cardiovascular risk reduction does not provide readily identifiable benefits to African Americans with hypertensive nephrosclerosis.

Aprepitant is an FDA-approved medication for the treatment of nausea following chemotherapy or surgery. Aprepitant is also used by some doctors to reduce nausea and vomiting in patients with gastroparesis, although the drug is not approved for this use. The Aprepitant for the Relief of Nausea in Patients with Chronic Nausea and Vomiting of Presumed Gastric Origin (APRON) study is a multicenter, randomized, placebo-controlled clinical trial designed to test the safety and efficacy of aprepitant in reducing symptoms among patients with gastroparesis. 

Patients at least 18 years of age who have experienced symptoms of chronic nausea and vomiting related to gastroparesis for at least 6 months are eligible for the study. Participants are randomized to treatment with either aprepitant (125 mg/day) or placebo for 4 weeks. Nausea symptoms are measured daily on a 0 to 100 mm visual analog scale (VAS) for one week at baseline and for four weeks after randomization. The primary outcome measure is improvement in VAS scores from baseline to through the course of treatment, as defined by a 25 mm or more reduction in mean VAS or attaining a mean VAS during the treatment period of < 25 mm. Improvement in other gastrointestinal symptoms, including symptoms of fullness, hunger, bloating, and abdominal pain, will be measured using the Gastroparesis Cardinal Symptom Index Daily Diary (GCSIDD), an overall relief of symptom questionnaire, and patient perception of improvement. Safety will be determined through satiety tests, electrogastrography for measuring of EGG dysrhythmias, and monitoring of side effects.

Sodium bicarbonate may help prevent kidney failure in people with chronic kidney disease. However, the dose to prescribe in order to test this possibility in a clinical trial is uncertain. The BASE pilot clinical trial will help determine the best dose of sodium bicarbonate to prescribe in a future study that will test the long-term safety and efficacy of sodium bicarbonate as a treatment to preserve kidney function in individuals with chronic kidney disease.

Urge incontinence, the involuntary urine loss associated with a strong desire to void, is one of several types of urinary incontinence (UI) and has one of the greatest impacts on quality of life. Common treatments for urge incontinence include behavioral training and antimuscarinic medications, but long-term adherence with medication therapy can be difficult to achieve. The Behavior Enhances Drug Reduction of Incontinence (BE-DRI) study was a multicenter, randomized clinical trial that combined behavioral training and pharmacological treatment with the goal of improving patient outcomes. The primary aim of this randomized clinical trial was to determine whether combining antimuscarinic drug therapy with supervised behavioral training, compared to drug therapy alone, improves the ability of women with urge incontinence to achieve clinically important reductions in incontinence episodes and to sustain these improvements after discontinuing medication. 

Participants with urge incontinence (defined as urge symptom index > stress symptom index on the Medical, Epidemiological, and Social Aspects of Aging Questionnaire) were enrolled in the study and randomly assigned either to drug therapy alone or combined behavioral and drug therapy. Drug therapy was tolterodine tartrate at a dose of 4 mg per day. Combination therapy included drug and behavioral training, which consisted of teaching pelvic floor muscle control and exercises, delayed voiding to increase voiding interval, individualized fluid management, and strategies to diminish urgency, suppress bladder contractions, and prevent both stress and urge incontinence. Treatment was discontinued for both groups after 10 weeks. The primary outcome measure, assessed at 8 months, was no receipt of drugs or other therapy for urge incontinence and a 70% or greater reduction in frequency of incontinence episodes. Secondary outcome measures included reduction in incontinence, self-reported satisfaction and improvement, and scores on validated questionnaires measuring symptom distress and health-related quality of life.

Results showed that the rate of successful discontinuation of therapy at 8 months was the same in the combination therapy and drug therapy alone groups, although a higher proportion of participants who received combination therapy than drug therapy alone achieved a 70% or greater reduction in incontinence at 10 weeks. The study concluded that the addition of behavioral training to drug therapy may reduce incontinence frequency during active treatment but does not improve the ability to discontinue drug therapy and maintain improvement in urinary incontinence.

Cystic fibrosis is an inherited condition in which cell secretions are thick and sticky, instead of thin and slippery. The secretions obstruct passageways and ducts, leading to severe internal damage. Patients with cystic fibrosis sometimes develop advanced liver disease (CFLD). Hepatic cirrhosis can occur with CFLD, as a result of the inflammation and scarring caused by clogged bile ducts.

The Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis Liver Disease (CFLD PUSH) study was a prospective longitudinal study that aimed to determine the utility of abdominal ultrasound to predict the development of cirrhosis in patients with cystic fibrosis as indicated by a nodular liver on ultrasound. Participants underwent abdominal ultrasound at enrollment and based on the outcome, were placed in one of four groups. Within a 10-year period, participants underwent other specimen collection procedures in addition to abdominal ultrasound. This study also monitored the effects of cystic fibrosis on associated pulmonary and nutritional issues.

The CIT consortium conducted a total of 9 studies across North America (CIT02 through CIT08) and the Nordic region (CIT01). CIT08 was a long-term follow-up study for interested participants at the North American sites. The target population is individuals with type 1 diabetes, normal kidney function, and intractable hypoglycemia. All studies treated participants with up to 3 separate infusions of islets. CIT01 participants were randomized to study treatment including Low Molecular Weight Sulfated Dextran (LMW-DS) and study treatment without LMW-DS. Subjects in both arms received induction and maintenance immunosuppression consisting of ATG (basiliximab instead of ATG for the 2nd and 3rd transplants, if applicable), etanercept, sirolimus, and tacrolimus. CIT01 subjects randomized to the LMW-DS arm also received LMW-DS before and during islet transplant.

The CIT consortium conducted a total of 9 studies across North America (CIT02 through CIT08) and the Nordic region (CIT01). CIT08 was a long-term follow-up study for interested participants at the North American sites. The target population was individuals with type 1 diabetes, normal kidney function, and intractable hypoglycemia. All studies treated participants with up to 3 separate infusions of islets. The CIT02 protocol focused on treating islets pre-transplant, and subjects were randomized to receive islets prepared with either Lisofylline or Exenatide. For induction and maintenance immunosuppression, the subjects received rabbit anti-thymocyte globulin (ATG; basiliximab instead of ATG for the 2nd and 3rd transplants, if applicable), etanercept, sirolimus, and tacrolimus in an open-label fashion.

The CIT consortium conducted a total of 9 studies across North America (CIT02 through CIT08) and the Nordic region (CIT01). CIT08 was a long-term follow-up study for interested participants at the North American sites. The target population was individuals with type 1 diabetes, normal kidney function, and intractable hypoglycemia. All studies treated participants with up to 3 separate infusions of islets. The focus of CIT04 was to evaluate the effectiveness of an IS regimen that included belatacept. For immunosuppression, CIT04 subjects received belatacept, basiliximab, and mycophenolate mofetil in an open-label fashion.

The CIT consortium conducted a total of 9 studies across North America (CIT02 through CIT08) and the Nordic region (CIT01). CIT08 was a long-term follow-up study for interested participants at the North American sites. The target population was individuals with type 1 diabetes, normal kidney function, and intractable hypoglycemia. All studies treated participants with up to 3 separate infusions of islets. Subjects in CIT05 received, in an open label-fashion, the anti-CD20 agent rituximab in combination with rabbit anti-thymocyte globulin (ATG; daclizumab or basiliximab instead of ATG for the 2nd and 3rd transplants, if applicable) for induction and sirolimus alone for maintenance, a calcineurin-inhibitor free regimen.

The CIT consortium conducted a total of 9 studies across North America (CIT02 through CIT08) and the Nordic region (CIT01). CIT08 was a long-term follow-up study for interested participants at the North American sites. The target population was individuals with type 1 diabetes, normal kidney function, and intractable hypoglycemia. All studies treated participants with up to 3 separate infusions of islets. Unlike the other studies, subjects in CIT06 had previously received a kidney transplant and were receiving maintenance immunosuppression. CIT06 was a single arm Phase 3 license-supporting study. At the time of islet transplant, subjects in CIT06 received induction therapy in an open-label fashion with rabbit anti-thymocyte globulin (ATG, 5 doses; daclizumab or basiliximab instead of ATG for the 2nd and 3rd transplants, if applicable) and etanercept. Subjects remained on their calcineurin-based maintenance immunosuppression regimen already in place for their renal allograft.

The CIT consortium conducted a total of 9 studies across North America (CIT02 through CIT08) and the Nordic region (CIT01). CIT08 was a long-term follow-up study for interested participants at the North American sites. The target population is individuals with type 1 diabetes, normal kidney function, and intractable hypoglycemia. All studies treated participants with up to 3 separate infusions of islets. Subjects in CIT07, a single arm Phase 3 license-enabling study, received induction and maintenance immunosuppression in an open-label fashion consisting of rabbit anti-thymocyte globulin (ATG; basiliximab instead of ATG for the 2nd and 3rd transplants, if applicable), etanercept, sirolimus and low-dose tacrolimus.

Phosphorus, a mineral found in many foods, works with calcium and vitamin D to keep bones healthy. Healthy kidneys keep the right balance of phosphorus in the body. Fibroblast growth factor 23 (FGF23) is a hormone that helps the kidneys to control the blood levels of phosphorus. 
 
In people with chronic kidney disease, kidneys struggle to get rid of enough phosphorus, so FGF23 levels increase in the blood to help get rid of extra phosphorus. Over time, with progressive loss of kidney function, both phosphorus and FGF23 can build up in the blood simultaneously. Researchers believe that too much phosphorus and FGF23 in the blood may lead to weak bones, increase the risk of heart problems, and accelerate progression of kidney disease. However, the best way to control phosphorus and FGF23 levels in the blood in patients with chronic kidney disease is not known.

COMBINE is a study to determine new methods to lower serum phosphorus levels and levels of FGF23 in chronic kidney disease patients.

The Chronic Prostatitis Collaborative Research Network (CPCRN), which consists of six clinical centers and one data coordinating center, was established to study all aspects of chronic prostatitis with the hope of improving diagnosis and treatment. Because the cause of chronic prostatitis/chronic pelvic pain syndrome (CP/CPPS) is unknown, affected men receive many empirical therapies. The two most common treatments prescribed by physicians are antimicrobial agents (including quinolones such as ciprofloxacin) and α-adrenergic receptor antagonists (such as tamsulosin), although there is little objective evidence to support their use. Based on these common clinical practices, the CPCRN conducted a randomized, two-by-two factorial design double-bind, clinical trial (CPCRN RCT#1) to evaluate whether ciprofloxacin or tamsulosin reduces symptoms of long-standing CP/CPPS of at least moderate severity. 

Men with CP/CPPS from 11 clinical centers were enrolled and randomly assigned in equal proportions to receive placebo; ciprofloxacin alone, 500 mg twice daily; tamsulosin alone, 0.4 mg once daily; or a combination of both drugs. Patients were treated for 6 weeks, at which time the primary endpoint was assessed. Symptoms at 9 and 12 weeks after randomization (3 and 6 weeks after completion of treatment) were also assessed to evaluate longer-term treatment response. The primary outcome measure was the change in the National Institutes of Health Chronic Prostatitis Symptom Index (NIH-CPSI) score from baseline to 6 weeks. Secondary outcome meaures included changes in the pain, voiding, and quality-of-life subscales of the NIH-CPSI; physical and mental summary scores on the Medical Outcomes Study 12-Item Short-Form Health Survey; and a 7-point patient-reported global response assessment. All adverse events were monitored and analyzed.

The study found that neither ciprofloxacin nor tamsulosin significantly reduced the NIH-CPSI total score over 6 weeks compared with placebo. Moreover, neither treatment showed a significant benefit among a wide range of secondary outcomes related to symptoms and quality of life. Findings do not support the use of these agents as empirical therapy for men with long-standing CP/CPPS and at least moderate symptoms.

The Chronic Renal Insufficiency Cohort (CRIC) Study is an observational study that examined risk factors for progression of chronic renal insufficiency (CRI) and cardiovascular disease (CVD) among CRI patients. The study enrolled adults aged 21 to 74 years with a broad spectrum of renal disease severity, half of whom were diagnosed with diabetes mellitus. Subjects underwent extensive clinical evaluation at baseline and at annual clinic visits and via telephone at 6 month intervals. Data on quality of life, dietary assessment, physical activity, health behaviors, depression, cognitive function, health care resource utilization, as well as blood and urine specimens were collected. Measures of kidney function and occurrence of new and worsening CVD  are the primary outcomes, among others.

Data from the recruitment and follow-up phases through Phase 3. Additionally, the data package includes variables related to APOL1, Echocardiogram, Electrocardiogram, and censored composite and individual outcomes. The data package includes data through 2024.

Retinal images for CRIC participants are not included in the package, but are available upon request.

Glomerular diseases are chronic diseases that cause damage to the filters, glomeruli, in the kidneys. Damaged glomeruli allow red blood cells and protein to leak into the urine, cause waste products to build up in the blood, and can lead to kidney failure. Glomerular diseases affect individuals of all ages and tend to progress slowly in many patients. Due to lengthy follow-up periods and challenges with patient recruitment, previous efforts to study glomerular diseases have been difficult.


Cure Glomerulonephropathy Network (CureGN) is a multi-center consortium that seeks to establish an infrastructure for the advancement of glomerular disease studies. CureGN recruits and maintains a large and diverse population of glomerular disease patients. The study established a database of patients diagnosed with the following conditions: minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), membranous nephropathy (MN), and immunoglobulin A nephropathy (IgAN). 

Currently, data from one analysis dataset is available for request.

Urinary tract infections (UTIs) are the most common serious bacterial infections in young children. When accompanied by a fever, there is an increased likelihood of a UTI also infecting the kidneys; between 15 and 52% of these children will develop kidney scarring as a result.

The Careful Urinary Tract Infection Evaluation (CUTIE) study was an observational study designed to determine why some children develop kidney scars after urinary tract infections (UTIs). The study was aimed at understanding which children were at the greatest risk of developing renal scarring following a UTI, so physicians can provide targeted therapies and appropriate interventions. Children between 2 months and 6 years of age who had a first or second UTI in the past 4 months were eligible for enrollment in the study. Participants were followed for 2 years through the course of five study visits, during which they were required to undergo a physical exam and provide information about recent medical history and quality of life via a questionnaire completed by parents. Additionally, blood and urine biospecimens were collected to monitor kidney function, and renal scans were performed to assess the development of kidney damage.
 
The CUTIE study was ancillary to the RIVUR (Randomized Intervention for Children with Vesicoureteral Reflux) study, which compared antibiotic prophylaxis to placebo in a cohort of children with a history of UTI and vesicoureteral reflux.  The CUTIE study focused specifically on children with a history of UTI who did not have vesicoureteral reflux. More information about the RIVUR study is available on the NIDDK repository website.

The Drug-Induced Liver Injury Network (DILIN) was established in 2003 as a cooperative agreement among the National Institutes of Health, 5 academic clinical centers, and a data coordinating center. A major emphasis of DILIN has been to establish a protocol for the identification and enrollment of patients with clinically significant DILI into a prospective observational study and to create a registry and specimen repository of biological samples that could be used for mechanistic studies on the etiology and prevention of DILI. <br><br>The DILIN prospective study is an ongoing multicenter observational study. Patients (2 years of age or older) were enrolled in this study if there was a strong clinical suspicion that a liver injury event was caused by a medication or an herbal agent occurring within 6 months before enrollment. Additionally, patients must meet one of the biochemical criteria for enrollment into this study. 

Data from the DILIN Prospective study through the grant 4 cycle (June 2023) are available from the Repository.

Drug-induced liver injury (DILI) is the single most common reason for regulatory actions concerning drugs, including failure to gain approval for marketing, removal from the market place, and restriction of prescribing indications. DILI is also a significant cause of morbidity and mortality in many patient populations. To stimulate and facilitate research into DILI, the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) has recently established the Drug-Induced Liver Injury Network (DILIN). One of the initial projects to be conducted by the network is to retrospectively establish a nationwide registry of patients who have suffered severe idiosyncratic liver injury associated with drugs (ILIAD), and to collect, and store serum, DNA from these patients (hereafter referred to as the “ILIAD protocol”). This ILIAD protocol will serve as a resource for subsequent mechanistic investigations of the basis for susceptibility to severe idiosyncratic DILI. 

Data from the DILIN Retrospective study through the grant 4 cycle (June 2023) are available from the Repository.

The Diabetes Prevention Program (DPP) was a multicenter trial examining the ability of an intensive lifestyle program or treatment with metformin to prevent or delay the development of type 2 diabetes in high-risk individuals with prediabetes. The DPP study showed that both interventions reduced the incidence of diabetes in participants, compared with placebo; the lifestyle intervention proved more effective than metformin in preventing the onset of diabetes. The Diabetes Prevention Program Outcomes Study (DPPOS) is the long-term follow-up of the original DPP study. The DPPOS sought to evaluate the effects of the interventions on the further development of diabetes and diabetes complications, including retinopathy, microangiopathy, and cardiovascular disease.

All active DPP participants were eligible for continued follow-up, and 88% of DPP participants enrolled in DPPOS (910 participants from the lifestyle, 924 from the metformin, and 932 from the original placebo groups). On the basis of the benefits from the intensive lifestyle intervention in the DPP, all three groups were offered group-implemented lifestyle intervention. Placebo was discontinued in the placebo group, metformin treatment was continued in the original metformin group, with participants unmasked to assignment, and the original lifestyle intervention group was offered additional lifestyle support. Comprehensive annual and semi-annual assessments similar to those done in the DPP study continued in DPPOS and included physical measurements, medical history updates, adverse event assessment, medication adherence and dispensing, questionnaires, and an annual oral glucose tolerance test (OGTT). OGTTs were discontinued after a confirmed diagnosis of diabetes.

DPPOS found that prevention or delay of diabetes with lifestyle intervention or metformin can persist for at least 10 years; the cumulative incidence of diabetes remained lowest in the lifestyle group.

Data collected after the final DPP Bridge visits in 2002 through February 2020 are available from the Repository.

Previous studies have shown an aggregation of Barrett’s esophagus (BE) and esophageal adenocarcinoma (EAC) (including esophagastric junctional adenocarcinoma [EGJAC]) in families, a syndrome that has been termed Familial Barrett’s esophagus (FBE). This aggregation, which suggests a biological relationship between BE and EAC, may be caused by common environmental exposures in family members, a genetic predisposition to disease, or both. For complex diseases such as BE and cancer, genetic influences may be more evident in pedigrees that have 3 or more affecteds. Thus, the Familial Barrett’s Esophagus study was established by the Familial Barrett’s Esophagus Consortium (FBEC) to determine whether cancers develop at an earlier age in multiplex Familial Barrett’s Esophagus (FBE) kindreds, defined as 3 or more members affected by Barrett’s esophagus (BE) or esophageal adenocarcinoma (EAC). Ultimately, the study aims to identify putative susceptibility genes for BE and its associated cancers. 

 Individuals with BE, EAC, or EGJAC were recruited at 8 academic care centers participating in the FBEC. After providing informed consent, participants were enrolled and given a self-administered FBE questionnaire. Information was collected on gastroesophageal reflux symptoms; risk factors such as age, gender, race, and self-reported obesity measures; and family history of BE, EAC, stomach and throat cancer (which may be incorrect ways of reporting esophageal cancer). Probands, defined as the first known affected member in a family, had the opportunity to discuss and clarify family history with their relatives before mailing back the questionnaire. Pedigrees were constructed from the questionnaire.  Differences in the age of diagnosis and other variables of interest, such as gender, obesity, race, acid regurgitation, heartburn, smoking and alcohol consumption, were assessed between the three family groups (non-familial, duplex and multiplex). Only probands and kindreds with a confirmed diagnosis were included in the analysis.

The study found that EAC is diagnosed at a significantly earlier age in multiplex FBE kindreds when compared to duplex and non-familial kindreds. Findings suggest that individuals who are members of multiplex FBE pedigree may be considered for screening at an earlier age than the general population. Following that earlier age of disease onset is considered an indicator of genetic susceptibility to disease, these results support a genetic basis for FBE. Future investigations into the genetic basis of FBE are needed and should focus on FBE kindreds with 3 or more members affected with defined BE and EAC phenotypes.

Functional dyspepsia (FD) is a common problem affecting up to 10-25% of individuals, and accounts for significant health care costs and affects quality of life. Currently there is no definitive treatment for FD, though antidepressants are used in the treatment of FD, but are considered unsatisfactory.  The Functional Dyspepsia Treatment Trial (FDTT) aims to test whether treatment with an antidepressant (amitriptyline or escitalopram) leads to improvement of symptoms in patients with moderate to severe FD.

Prior observational studies have shown multiple benefits of both frequent hemodialysis (HD) and frequent nocturnal HD compared to conventional three times per week treatments for patients with end-stage renal disease (ESRD). To test these hypotheses, the Frequent Hemodialysis Network (FHN) Trials Group conducted two multicenter randomized trials to compare conventional tree times weekly HD with (1) in-center daily and (2) home nocturnal HD. Specifically, the FHN Nocturnal trial was designed to assess the effects of frequent nocturnal home HD six times per week compared with conventional three times per week HD.

Participants with ESRD requiring maintenance dialysis were recruited from 19 clinical centers. A total of 87 subjects were enrolled, underwent baseline assessment, and were randomized to treatment, either to three times per week conventional HD or to nocturnal HD six times per week, all with single-use high-flux dialyzers. Participants were followed for 14 months. The composite of mortality with change in (1) left ventricular mass index (LVMI) by magnetic resonance imaging (MRI), and (2) SF-36 RAND Physical Health Composite (PHC) were used as co-primary outcomes to assess the efficacy of treatment.  Secondary outcomes included cognitive performance, self-reported depression, laboratory markers of nutrition, mineral metabolism and anemia, blood pressure and rates of hospitalization, and vascular access interventions.

The FHN Nocturnal trial found no significant effect of nocturnal hemodialysis for either of the two co-primary outcomes. Results showed that patients in the nocturnal arm had improved control of hyperphosphatemia and hypertension, but there was no significant benefit to frequent nocturnal hemodialysis among the other main secondary outcomes. While the nocturnal study was unable to demonstrate a definitive benefit of more frequent nocturnal hemodialysis for either co-primary outcome, these results differ from the FHN Daily Trial, which showed a statistically significant benefit of more frequent hemodialysis for both co-primary outcomes.
The primary objective of the FHN Nocturnal trial was to compare the effects of conventional (three times per week) and frequent (six times per week) hemodialysis treatment for patients with ESRD, specifically to assess the safety, efficacy, and cost-effectiveness of the two treatments.

The Family Investigation of Nephropathy and Diabetes (FIND) was a multicenter observational study designed to identify genetic genetic determinants of diabetic kidney disease. Study participants were recruited from 11 centers and in many ethnic groups throughout the United States for a genome-wide association study (GWAS). Two additional strategies were also used to localize susceptibility genes: a family-based linkage study and a case-control study using mapping by admixture linkage disequilibrium (MALD). In the family-based study, participants with diabetic nephropathy were recruited with their parents and selected siblings. Linkage analyses were conducted to identify chromosomal regions containing genes that influence the development of diabetic nephropathy and related quantitative traits.  Subsequently, a GWAS was carried out.

Two types of MALD studies were done. The first was a case-control study of unrelated individuals of Mexican American heritage in which both cases and controls had diabetes, but only the case had nephropathy. The second was a case-control study of African American patients with nephropathy (cases) and their spouses (controls) unaffected by diabetes and nephropathy; offspring were genotyped when available to provide haplotype data. Through the family and MALD studies, the FIND study identified several genes that contribute to diabetic nephropathy, albuminuria, and glomerular filtration rate (GFR), including ApoL1.

Note: Native American participants have been excluded due to consent to not share data.

The Dialysis Access Consortium (DAC) was developed to investigate interventions to improve hemodialysis vascular access outcomes in patients with end-stage renal disease (ESRD). The Clopidogrel Prevention of Early AV Fistula Thrombosis (FISTULA) study was one of two major clinical trials conducted under the DAC.  The study was a randomized, double-blind, placebo-controlled trial of clopidogrel administered following fistula creation in a large group of patients with advanced chronic kidney disease or ESRD. The arteriovenous fistula is the preferred type of vascular access for hemodialysis because of lower thrombosis and infection rates and lower health care expenditures compared with synthetic grafts or central venous catheters; however, early failure of fistulas due to thrombosis or inadequate maturation is a barrier to increasing the prevalence of fistulas among patients treated with hemodialysis. The FISTULA study aimed to investigate the ability of platelet inhibition to prevent early fistula failure and increase the number of fistulas that ultimately become usable for hemodialysis access.

Participants with chronic kidney disease or ESRD who underwent creation of a new native arteriovenous (AV) fistula were randomized to treatment with clopidogrel or placebo for six weeks following fistula creation surgery.  The primary outcome was fistula thrombosis, determined by physical examination at 6 weeks. The secondary outcome was failure of the fistula to become suitable for dialysis. The study found that clopidogrel reduces the frequency of early thrombosis of new arteriovenous fistulas but does not increase the proportion of fistulas that become suitable for dialysis.

Nonalcoholic fatty liver disease (NAFLD) is characterized by hepatic steatosis in the absence of a history of significant alcohol use or other known liver disease. Nonalcoholic steatohepatitis (NASH) is the progressive form of the disease, which results in cirrhosis, liver failure, and death in a substantial proportion of patients. Treatment approaches to NAFLD in adults and children target reduction in insulin resistance and oxidative stress. Several agents have been found to be promising as therapy of NAFLD in small clinical trials, but none have been proven to alter its natural history or outcome, and none are approved for general use in this disorder.  The Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) was established by the NIDDK in 2002 to investigate the natural history, pathogenesis, and therapy of NAFLD and NASH in both children and adults. The Farnesoid X Receptor (FXR) Ligand Obeticholic Acid in Nonalcoholic Steatohepatitis (NASH) Treatment (FLINT) trial was a multicenter, randomized, placebo-controlled clinical trial conducted by the NASH CRN to investigate the safety and efficacy of treatment of obeticholic acid compared to placebo in adults with nonalcoholic fatty liver disease.

Individuals with histologic evidence of NASH, based on a liver biopsy and a disease activity score (NAS) of at least 4 (with a score of at least 1 in each component of the NAS 8-point scale), were enrolled and randomized to treatment with either obeticholic acid (25 mg) or placebo for 72 weeks. Each participant received a baseline oral glucose tolerance test and a baseline MRI scan to determine liver fat content. Additionally, information was collected on demographics, alcohol consumption, and medication use. The primary outcome measure was improvement in the NAS score by at least 2 points with no worsening of liver fibrosis and was assessed by liver biopsy at 72 weeks. Secondary outcome measures included change in NASH diagnosis (from determinate or indeterminate to not-NASH), fibrosis score, hepatocellular ballooning score, hepatic fat fraction, serum aminotransferase and gamma-glutamyl transpeptidase (GGT) levels, fasting glucose levels, and health related quality of life scores.

A planned interim analysis of end-of-treatment liver biopsies from 58% (164/283) of enrolled patient demonstrated that the primary endpoint had been reached and the statistical significance crossed the preset boundry set for efficacy (p ≤ 0.0031). Accordingly, further liver biopsies were not performed. Additionally, because of the observation of disproportionate lipid abnormalities among patients receiving OCA and in the context of the study having met its primary endpoint, further treatment with OCA and placebo was discontinued and remaining patients completed a 24-week follow-up off treatment.

The CIT consortium conducted a total of 9 studies across North America (CIT02 through CIT08) and the Nordic region (CIT01). CIT08 was a long-term follow-up study for interested participants at the North American sites. The target population was individuals with type 1 diabetes, normal kidney function, and intractable hypoglycemia. All studies treated participants with up to 3 separate infusions of islets. The focus of CIT03 was to evaluate the investigational agent, deoxyspergualin. Subjects in CIT03 received rabbit anti-thymocyte globulin (ATG; basiliximab instead of ATG for the 2nd and 3rd transplants, if applicable), deoxyspergualin, etanercept, sirolimus, and low-dose tacrolimus in an open-label fashion.

The Acute Liver Failure Study Group (ALFSG) was a clinical research network that collected data and biospecimens from participants with acute liver failure (ALF) and acute liver injury (ALI). ALF is defined as a severe form of acute liver injury characterized by rapid onset over days and weeks in the absence of underlying cirrhosis, leading to abnormal coagulation with prolongation of the prothrombin time (INR ≥ 1.5) in the presence of altered mentation. ALI is defined as a severe acute liver injury with an INR ≥ 2.0 and no encephalopathy (altered mental functioning, drowsiness, or coma). Outcomes vary, but nearly 29% die while another 22% undergo liver transplantation, and the remaining recover fully in general. A unique feature of ALF and ALI is that there are a variety of causes, all of which share similar clinical features (coagulopathy, encephalopathy, susceptibility to infection, and bleeding) regardless of cause. The most frequent causes are acetaminophen overdose, drug-induced liver injury, autoimmune hepatitis, and viral hepatitis B, but less common causes include acute fatty liver of pregnancy, ischemia/shock, Wilson disease, heat stroke, and metastatic cancer. 

ALFSG conducted a Multi-Center Trial to Study Adult Acute Liver Failure (AALF) with ALF and ALI participants and those less severe that had coagulopathy but not the threshold of encephalopathy, a pilot study with a subset of AALF participants using rotational thromboelastography as a dynamic measure of coagulation, and a clinical trial with a subset of AALF participants using 13C labeled methacetin breath testing to determine outcomes in ALF patients being considered for transplantation.

When traditional approaches for weight loss, such as diet, exercise, and medications, fail as effective treatments for obesity, bariatric surgical procedures that restrict stomach size or lead to altered absorption of nutrients may be performed. While these procedures often result in significant, sustained weight loss, the benefits must be balanced against the short- and long-term risks of bariatric surgery. The Longitudinal Assessment of Bariatric Surgery (LABS) observational study was established to assess the risks and health benefits associated with bariatric surgery and to identify aspects of the procedures and patient characteristics associated with optimal outcomes. The study was organized into three phases: LABS-1, which evaluated the short-term safety of bariatric surgery; LABS-2, which evaluated the longer-term safety and efficacy of bariatric surgery, and examined patient characteristics associated with short- and intermediate-term outcomes; and LABS-3, which examined psychosocial, quality of life, and health economics aspects of obesity and the mechanisms of obesity-related diseases.

The LABS-1 study enrolled participants at least 18 years of age who underwent bariatric surgical procedures between March 2005 and December 2007 at LABS surgical centers. Participants underwent a pre-operative evaluation, during which data was collected on height, weight, blood pressure, comorbid conditions, and physical examination. Participant outcomes were recorded post-operatively.

An ancillary study from LABS-1 regarding psychosocial issues associated with weight loss was conducted at two of the LABS study centers, and a set of control participants were recruited from primary care practices outside of the LABS consortium. The overall duration of the ancillary study was 48 months with the goal of assessing changes in sexual function and sex hormones in the two groups at predetermined follow-up visits.

An ancillary study from LABS-1 regarding cognitive effects of bariatric surgery was conducted at two of the LABS study centers, and a set of control participants were recruited who did not plan to have bariatric surgery for at least two years. The overall duration of the ancillary study was 24 months with a subset of participants providing additional follow-up for 48 months.

Both the ancillary studies and full data for LABS-1 are available in the data package.

The TODAY2 study was a longitudinal follow-up study to the TODAY (Treatment Options for Type 2 Diabetes in Adolescents and Youth) study that continued the care and observation of the TODAY cohort participants beyond the end of the TODAY intervention trial. The TODAY2 study had two phases: 1) Transition of TODAY participants to non-blinded, non-randomized standard diabetes care and management with monitoring and follow-up for up to 24 months, and 2) Long-term longitudinal follow-up of the TODAY cohort based on findings from TODAY.

The Adult-to-Adult Living Donor Liver Transplantation Cohort Study (A2ALL) was a consortium of 9 U.S. liver transplant centers performing adult-to-adult living donor liver transplant (AALDLT) with the primary goal of examining outcomes of AALDLT versus deceased donor liver transplant (DDLT).  AALDLT is a relatively new procedure increasingly used at major transplantation centers. Relatively small numbers of cases are performed at any one center and approaches to the patient and donor are too diverse across centers to provide reliable and generalizable information on donor and recipient outcomes from individual centers. Therefore, the consortium was organized to accrue and follow sufficient numbers of patients being considered for and undergoing AALDLT to provide generalizable results from adequately powered studies.

The Adult to Adult Living Donor Liver Transplantation Cohort Study (A2ALL) consisted of both retrospective and prospective studies of AALDLT. Legacy data from the A2ALL retrospective cohort is housed in the NIDDK Data Repository. The A2ALL retrospective cohort comprised 819 adult patients, each with a potential living donor, evaluated between January 1, 1998 and February 28, 2003. The retrospective study was designed to gain initial insights into outcomes associated with liver transplant procedures, with data drawn from existing medical records and patient materials. It was hypothesized that pursuit of a living liver allograft leads to decreased pre-transplant morbidity and mortality and better long term outcomes for patients starting from the point at which listed patients have a potential donor evaluated (at least a history and physical examination). The study analyzed numerous variables to determine the factors influencing allograft survival in recipients.
 
The A2ALL study showed evidence that AALDLT is a viable option for liver replacement with outcomes that improve with center experience. Donor characteristics associated with survival included younger age and cold ischemia time of the right lobe graft. 

MRI and CT Scan images from subjects enrolled in A2ALL are not included in the data package, but are available upon request., EDIT The Adult-to-Adult Living Donor Liver Transplantation Cohort Study (A2ALL) was a consortium of 9 U.S. liver transplant centers performing adult-to-adult living donor liver transplant (AALDLT) with the primary goal of examining outcomes of AALDLT versus deceased donor liver transplant (DDLT).  AALDLT is a relatively new procedure increasingly used at major transplantation centers. Relatively small numbers of cases are performed at any one center and approaches to the patient and donor are too diverse across centers to provide reliable and generalizable information on donor and recipient outcomes from individual centers. Therefore, the consortium was organized to accrue and follow sufficient numbers of patients being considered for and undergoing AALDLT to provide generalizable results from adequately powered studies.

The Adult to Adult Living Donor Liver Transplantation Cohort Study (A2ALL) consisted of both retrospective and prospective studies of AALDLT. Legacy data from the A2ALL retrospective cohort is housed in the NIDDK Data Repository. The A2ALL retrospective cohort comprised 819 adult patients, each with a potential living donor, evaluated between January 1, 1998 and February 28, 2003. The retrospective study was designed to gain initial insights into outcomes associated with liver transplant procedures, with data drawn from existing medical records and patient materials. It was hypothesized that pursuit of a living liver allograft leads to decreased pre-transplant morbidity and mortality and better long term outcomes for patients starting from the point at which listed patients have a potential donor evaluated (at least a history and physical examination). The study analyzed numerous variables to determine the factors influencing allograft survival in recipients.
 
The A2ALL study showed evidence that AALDLT is a viable option for liver replacement with outcomes that improve with center experience. Donor characteristics associated with survival included younger age and cold ischemia time of the right lobe graft. 

MRI and CT Scan images from subjects enrolled in A2ALL are not included in the data package, but are available upon request.

Standard treatment for primary focal segmental glomerulosclerosis (FSGS) includes administration of corticosteroids and immunosuppressive therapy, with the goals of achieving complete remission of proteinuria and preservation of renal function.  When standard treatments fail to induce remission, a number of agents are used as renoprotective therapy to delay progression of chronic kidney disease (CKD) to end stage renal disease (ESRD); however, there is a need for the development of new therapies that are safe and effective for patients with FSGS, particularly those for whom standard therapy fails. The Novel Therapies for Resistant FSGS (FONT II) trial was a randomized, multicenter Phase I/II clinical trial designed to investigate the potential efficacy of novel therapies for resistant FSGS. Two different therapies, adalimumab (a human anti-TNF-α antibody) and galactose, were evaluated against standard conservative therapy for resistant FSGS, which consists of the combination of lisinopril, losartan, and atorvastatin. Additional aims of the study included identification of one or more novel agents as candidates for evaluation in a future Phase III trial and creation of an infrastructure for the timely completion of clinical trials in patients with rare glomerular diseases such as FSGS. 

Individuals with primary FSGS confirmed by renal biopsy who failed to respond to prior immunosuppressive therapy were eligible for the study. Participants were required to complete a run-in period in which they were taken off all immunosuppressive medications, including corticosteroids, for 30 days. Participants who achieved an estimated GFR ≥ 30 mL/min/1.73 m2 at the end of the run-in period were randomized to treatment with adalimumab, galactose, or standard conservative therapy. The primary outcome measure, evaluated at 6 months, was a composite of two end points, a reduction of proteinuria by 50% from the baseline value and an estimated GFR (eGFR) that was either ≥ 75% of the value at time of randomization or that was persistently ≥ 75 mL/min/1.73 m2 in those whose renal function was ≥ 75 mL/min/1.73 m2. Adverse effect profile, patient satisfaction score using the TSQM questionnaire, percent change in proteinuria (evaluated as a continuous variable) and change in or time to doubling of eGFR were also assessed as secondary outcome measures.

For diabetics with gastroparesis, unpredictable gastric emptying rates and uncertainties in nutrient absorption can lead to difficulties in managing glycemia. The utility of continuous glucose monitoring (CGM), which quantifies real time glucose excursions, in planning periprandial insulin dosing for diabetic patients with gastroparesis has not been explored. The Safety, Feasibility, and Potential Efficacy of Continuous Glucose Monitoring and Insulin Pump Therapy in Diabetic Gastroparesis (GLUMIT-DG) study is a multicenter, uncontrolled clinical trial that was established by the Gastroparesis Clinical Research Consortium (GpCRC) to assess the safety and utility of CGM in guiding insulin pump therapy in patients with type 1 and type 2 diabetes and gastroparesis. 

Individuals with symptoms of gastroparesis, a Gastroparesis Cardinal Symptom Index (GCSI) score of 18, and diabetes for at least 2 years are enrolled in the study. Participants are treated using a combination of CGM and insulin pump therapy. The primary outcome measure, the frequency of mild and moderate (glucose level < 70 mg/dL) and severe hypoglycemic episodes while undergoing therapy, is assessed at 12 and 24-week follow-up visits. CGM data are related to finger stick levels and HbA1c values to test comparability of CGM and finger stick values. The efficacy of CGM-guided insulin pump therapy on symptoms of gastroparesis, quality of life, satiety, gastric myoelectrical activity, and autonomic function are also evaluated as a secondary outcome measures.

The Genetics of Kidneys in Diabetes (GoKinD) study was an initiative that aimed to identify genes that are involved in diabetic nephropathy. A large number of individuals with type 1 diabetes were screened to identify two subsets, one with clear-cut kidney disease and another with normal renal status despite long-term diabetes. Those who met additional entry criteria and consented to participate were enrolled. When possible, both parents also were enrolled to form family trios. DNA, serum, plasma, and urine samples and clinical information were collected from participants. A repository of genetic and clinical information was created for use in genetic analysis to investigate the genetic risk factors of the renal disease of Type 1 diabetes, with the ultimate aim of determining pathways for intervention to prevent diabetic kidney disease and allow targeting of preventive therapies.

GWAS data must be requested through dbGAP.

Gastroparesis, a syndrome in which patients experience delayed gastric emptying, is characterized by symptoms such as nausea, vomiting, bloating, abdominal pain, and early satiety. While gastroparesis can present as a complication of diabetes and gastric surgery, the underlying cause is unknown in many cases. Despite the significant morbidity of the condition, research and characterization of the clinical features of gastroparesis are limited, hindered by challenges such as single centers and small study populations. The Gastroparesis Registry is an observational study created by the Gastroparesis Clinical Research Consortium (GpCRC) to enroll a sufficiently large number of patients to elucidate the etiology and clinical features of the condition. 

Individuals with symptoms of gastroparesis of at least 12 weeks duration, delayed gastric emptying on scintigraphy, and no abnormality causing obstruction on upper endoscopy were enrolled. Data was collected on medical history, physical examinations, upper endoscopy results, and 4 hour gastric emptying scintigraphy results. Symptoms of gastroparesis and psychological functioning were assessed using patient-completed questionnaires, including the Patient Assessment of Upper Gastrointestinal Disorders Symptoms Severity Index (PAGI-SYM), the Beck Depression Inventory (BDI), and the State-Trait Anxiety Inventory (STAI). Laboratory tests were obtained to measure complete blood count with erythrocyte sedimentation rate (ESR) and C reactive protein (CRP).

Analyses were done to examine such baseline characteristics as demographics, lifestyle, co-morbidities, laboratory values, psychological evaluation test scores, gastroparesis-specific medical history, gastric emptying scintigraphy results, and symptom severity among all patients and among various patient sub-groups. Comparisons were also done to evaluate the similarities and differences of the diabetic and idiopathic presentations of gastroparesis. Results showed that IG is a diverse syndrome that varies by sex, body mass, symptom onset, and delay in gastric emptying. Following conclusion of the GpR study, a follow-up study was initiated to expand the registry (GpR 2).

The Dialysis Access Consortium (DAC) was developed to investigate interventions to improve hemodialysis vascular access outcomes in patients with end-stage renal disease (ESRD). The Aggrenox Prevention of Access Stenosis Study (GRAFT) was one of two major clinical trials conducted under the DAC.  Surgically created arteriovenous (AV) grafts are the most common type of hemodialysis vascular access in the United States, but fail frequently due to the development of venous stenosis. The GRAFT study was a double-blind, placebo-controlled randomized clinical trial designed to test whether treatment with Aggrenox (dipyridamole and low-dose aspirin) prolonged the primary unassisted patency of newly placed grafts. Participants undergoing placement of a new AV graft for hemodialysis were randomly assigned to receive treatment with Aggrenox or placebo immediately following access surgery. The primary outcome measure was loss of primary unassisted patency, defined as the time from access placement until thrombosis or requirement for intervention to maintain or restore patency. Major secondary outcome measures included cumulative access failure and death. The GRAFT study found that treatment with Aggrenox had a significant but modest effect in reducing the risk of stenosis and improving the duration of primary unassisted patency of newly created grafts.

The Hepatitis C Antiviral Long-Term Treatment Against Cirrhosis Trial (HALT-C) was a randomized clinical trial of long-term use of peginterferon alfa-2a in patients with chronic hepatitis C. The study investigated whether long-term antiviral therapy could prevent progressive liver disease—including cirrhosis, liver failure, hepatocellular carcinoma, and death—in chronic hepatitis C participants. Participants with chronic hepatitis C and advanced fibrosis who failed to respond to prior treatment with peginterferon and ribavirin were enrolled in the study. The participants, who were stratified according to stage of fibrosis, were randomly assigned to receive either peginterferon or no therapy for 3.5 years. The primary outcome measure was progression of liver disease, as indicated by death, hepatocellular carcinoma, hepatic decompensation, or, for those with bridging fibrosis at baseline, an increase in the Ishak fibrosis score of 2 or more points. Results showed that the level of serum aminotransferases, the level of serum hepatitis C virus RNA, and histologic necroinflammatory scores all decreased significantly with treatment, but there was no significant difference between the groups in the rate of disease progression.

Some of the patients with hepatitis C-related advanced liver disease developed hepatocellular carcinoma (HCC), and there is an existing matched set of samples from these patients and controls. Please see the HCC Documents detailing the description of this set of samples and guidelines for its use in validating serum biomarkers. Investigators interested in using this set should make sure to refer to it specifically in their requests.

Hepatitis B, a significant cause of cirrhosis and hepatocellular carcinoma worldwide, affects an estimated 800,000 to 1.4 million people in the United States. While progress has been made in the prevention, diagnosis, and treatment of chronic hepatitis B, challenges remain in identifying persons affected by the virus and in determining recommendations for management and treatment. The Hepatitis B Research Network (HBRN) was a multicenter network to investigate the etiology and progression of the disease and to test the safety and efficacy of current treatment approaches. The HBRN Adult Cohort Study (HBRN Cohort A) was designed to describe participants with hepatitis B virus (HBV) infection and identify predictors of disease activation and progression. The relationship of HBV genotype to clinical, biochemical, and histological characteristics and to pregnancy was also explored.

Individuals of at least 18 years of age that were hepatitis B surface antigen (HBsAg) positive were enrolled in the HBRN Adult Cohort study. Baseline data were collected on demographics, medical history, family history of liver disease, and health behaviors. Participants were categorized into various phases of HBeAg-positive and HBeAg-negative HBV infection and stages of HBV disease for monitoring of changes in HBV infection status and quantitative HBsAg levels. The rate of various clinical outcomes—including hepatitis exacerbation marked by alanine aminotransferase (ALT) flare, antigen loss of HBsAg or HBeAg, cirrhosis, development of hepative decompensation, heptaocellular carcinoma (HCC), death, and liver transplantation—and the factors associated with these outcomes were assessed as primary outcome measures, evaluated at up to 288 weeks. Analyses were performed to determine whether a baseline HBsAg below 1,000 IU/mL and HBV DNA below 1,000 IU/mL were accurate predictors of people who were inactive carriers (defined as people who were HBsAg positive, HBeAg negative, had normal ALT and HBV DNA under 1,000 IU/mL on at least two occasions over a period of at least 6 months with HBV DNA under 1,000 IU/mL). Additionally, biospecimens were collected from participants to create a repository of resources for future studies.

The HEALTHY study is a randomized prevention trial that investigated the effectiveness of an integrated intervention in middle schools that aimed to moderate risk for type 2 diabetes in children. The trial was conducted at 7 field centers in 42 middle schools, randomly assigned to intervention or control. Student recruitment and baseline data collection occurred during in the first semester of 6th grade (2006); the intervention was implemented the following semester (2007) and continued through 8th grade (2007-2009). For schools that received intervention, the program consisted of four integrated components targeting nutrition, physical education, behavior change initiatives, and educational communications activities that were designed to impact the environment and lifestyle choices of middle school children. The intervention was implemented either school-wide or grade-wide, so all students were exposed; those who provided appropriate informed consent participated in data collection and evaluation. The primary outcome measure was the combined prevalence of overweight and obesity (BMI ? 85th percentile). The study found that the combined prevalence of overweight and obesity decreased in both intervention and control schools, with no significant difference between school groups. However, the intervention did result in significantly greater reductions in various indexes of adiposity, including obesity.

Detailed information about the HEALTHY study, including the protocol, intervention materials, and publications, is available at <a href="//www.healthystudy.org.">www.healthystudy.org.</a> <a class="ExternalLinkIcon_externalLink__tu8Oz" href="https://www.niddk.nih.gov/disclaimers" target="_blank" rel="noopener noreferrer"> <svg aria-label="This link is external" xmlns="http://www.w3.org/2000/svg" width="10" height="10" fill="currentColor" class="bi bi-box-arrow-up-right" viewBox="0 0 16 16">
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In patients undergoing hemodialysis for end-stage renal disease, a large number of fistulas fail to mature adequately to support the hemodialysis blood circuit and cannot be used for dialysis. Currently, hemodialysis access using fistulas is hindered by a lack of predictors of and therapies for arteriovenous fistula (AVF) failure.  The Hemodialysis Fistula Maturation Study was a prospective, multicenter observational study that was established by the Hemodialysis Fistula Maturation Consortium (HFMC) to identify the clinical and biological factors associated with AVF maturation outcomes. The primary objectives of the study included improving prediction of AVF maturation and exploring the mechanism of AVF failure by examining the relationship between clinical usability and anatomy, biology, clinical attributes, and processes of care.

The study enrolled patients nearing or with end-stage renal disease who are undergoing AVF creation for hemodialysis. Data were collected pre-operatively, peri-operatively, and post-operatively. Medical history, demographics, and physical examination information were collected prior to surgery at baseline. Participants undergone preoperative ultrasound arterial and venous mapping, flow-mediated and nitroglycerin-mediated brachial artery dilation, arterial pulse wave velocity, and venous distensibility; additionally, ultrasounds will be administered at 1 day, 2 weeks, 6 weeks, and prior to fistula intervention and initial cannulation. Information were collected peri-operatively on surgical personnel, surgical procedures, and clinical observations. Outcome data on AVF usability and other dialysis-related data were collected post-operatively. In addition to these measurements, tissue and blood specimens were collected for analyses of histology, morphometry, immunohistochemistry, and gene expression.

The NIDDK Inflammatory Bowel Disease Genetics Consortium (IBDGC) and cell line repository was created in 2002 by the National Institute of Diabetes, Digestive and Kidney diseases (NIDDK) to advance knowledge on the inflammatory bowel diseases, specifically Crohn’s Disease and Ulcerative Colitis. The Consortium consists of six genetic research centers (GRC) and a data coordinating center (DCC) that prospectively recruits a combination of cases, controls, and trios to gather a large collection of samples and linked phenotype information. Phenotyping is performed using a standardized protocol, and lymphoblastoid cell lines are established for each subject. The resulting DNA samples are used to conduct genetic linkage and association studies. The IBDGC is involved with independent genetic research studies and actively works with members of the IBD and genetic communities on collaborative projects.

The Interstitial Cystitis Clinical Trials Group (ICCTG) RCT1 was a pilot clinical trial using a 2x2 factorial study design with double masked drug treatment to evaluate the feasibility of oral pentosan polysulfate (PPS) and oral hydroxyzine in patients with interstitial cystitis (IC).  IC is a bladder syndrome characterized as painful, debilitating and chronic, with no widely accepted effective treatment. Characteristic symptoms include pain with bladder filling, and marked urinary frequency (to relieve pain). The presentation of symptoms can be quite variable among patients, suggesting that IC is a multi-factorial syndrome with several proposed etiologies, some of which may be interrelated.
Subjects with IC were randomized over 18 months and followed for 24 weeks. The primary endpoint was a patient reported global response assessment (GRA) of overall symptoms. Secondary endpoints included symptom indexes and patient reports of urinary pain, urgency and frequency.  The low global response rates for PPS and hydroxyzine suggest that neither provided benefit for the majority of patients with IC.

The ICCTG RCT2 study was a randomized, placebo-controlled, two-arm clinical trial that investigated the efficacy and safety of intravesical Bacillus Calmette-Guerin (BCG) therapy in treating patients with treatment refractory Interstitial Cystitis (IC). IC is a bladder syndrome characterized as painful, debilitating and chronic, with no widely accepted effective treatment. Characteristic symptoms include pain with bladder filling, and marked urinary frequency (to relieve pain). The presentation of symptoms can be quite variable among patients, suggesting that IC is a multi-factorial syndrome with several proposed etiologies, some of which may be interrelated.

Subjects with IC were randomized to 6 weekly double-blinded intravesical instillations of either BCG or placebo, and then followed for a total of 34 weeks. The primary outcome was a patient reported global response assessment at week 34, supplemented with medications for IC during weeks 31 to 34 recorded in a diary. Although the safety profile for BCG was acceptable, the therapy did not show significant benefit compared to placebo.

Cholestasis, a rare condition involving a reduction or obstruction of bile flow from the liver to the small intestine, can cause significant growth problems, liver complications, the need for liver transplantation, and death. The four rare genetic disorders Alagille syndrome (ALGS), alpha-1 antitrypsin (a-1AT) deficiency, bile acid synthesis defects, and progressive familial intrahepatic cholestasis (PFIC) account for approximately 20% to 30% of all infant cases of cholestasis. Current knowledge concerning the etiology and outcomes of these diseases is limited. The Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC) study is a longitudinal cohort study that was established by the Childhood Liver Disease Research and Education Network (ChiLDReN) to investigate the natural history and progression of these four genetic disorders.  

Children and adults ages 6 months through 25 years who have been diagnosed with ALGS, a-1AT deficiency, bile acid synthesis defects, and PFIC are enrolled. Individuals who are siblings of a-A1T participants and have underlying disease with no evidence of liver involvement may also be enrolled. Participants will complete a baseline visit and give annual follow-up visits. Study visits involve review of clinical information, family history, and any treatments and their outcomes. A physical exam, laboratory tests, and radiologic and imaging evaluations will also be performed. In addition to these standard of care evaluations, participants will undergo neurodevelopment evaluations, DEXA scanning, hearing exams, and liver histology studies. The primary outcome measure is the evaluation of disease progression for each condition, which includes assessment of growth failure, worsening liver functions, developmental complications of portal high blood pressure, liver transplantation, and death. Jaundice, listing for liver transplantation, calculated pediatric end-stage liver disease (PELD) score or model for end-stage liver disease (MELD) score, health-related quality of life, growth, bone mineral density, and presence of hearing loss are assessed as secondary outcome measures. 

The data package now includes analysis datasets from multiple publications.

The Medical Therapy of Prostatic Symptoms (MTOPS) study was a multi-center, randomized, double-blind placebo controlled clinical trial that tested whether the oral drugs finasteride (Proscar) and doxazosin (Cardura), alone or in combination, could delay or prevent the worsening of symptoms in men with benign prostatic hyperplasia (BPH). Doxazosin is an α-adrenergic receptor agonist (α-blocker) that reduces muscle tone of the prostate and bladder neck, and finasteride is a 5-α-reductase inhibitor that reduces prostate volume by inducing epithelial atrophy. The MTOPS study hypothesized that these two classes of drugs may act synergistically to delay or prevent the clinical progression of BPH.
 
Participants were randomly assigned, in a double-blind fashion, to one of the following 4 treatment groups: placebo, doxazosin, finasteride, or combination therapy. The primary outcome measure was time to overall clinical progression of BPH, defined as either a confirmed 4 point or greater increase from baseline in the American Urological Association (AUA) symptom score, acute urinary retention, incontinence, renal insufficiency, or recurrent urinary tract infection. The progression of BPH was also assessed using digital rectal examination, serum PSA measurement, urinalysis, transrectal ultrasound, biopsies, urinary flow, and change in the AUA score. 
The study found that combination therapy with doxazosin and finasteride was more effective than either therapy alone in the preventing the clinical progression of BPH.

It has been proposed that the chronic, long-term ingestion of analgesics leads to specific renal pathology and renal failure. Previous studies suggested that the non–contrast-enhanced computerized tomography (CT) scan is a highly reliable tool for the diagnosis of analgesic-associated renal disease. The National Analgesic Nephropathy Study (NANS) is a case control study that was established to examine the association between heavy analgesic use and renal insufficiency and to investigate the ability of the CT scan to detect heavy analgesic use in the US population with end-stage renal disease (ESRD).

Patients with ESRD without a clear nonanalgesic cause for renal failure who had begun dialysis within the previous 3 months were enrolled in the study. A non-contract enhanced CT scan was performed using a standardized protocol. Three parameters—size, indentations, and calcifications—were used to identify patients who might have analgesic-induced kidney disease. Additionally, an interview was conducted to obtain information from patients concerning analgesic use, pre-dialysis signs and symptoms of renal disease, medical history, and demographics. 

The study concluded that findings of small indented calcified kidneys (SICK) did not occur with sufficient frequency in US patients with ESRD and heavy analgesic exposure to render the non-contrast-enhanced CT scan a sensitive tool to identify such individuals. Although the conclusion concerning the lack of diagnostic value of the CT scan is clear, the study did not address the overall relationship between analgesics and the progression of renal disease. Results showed an association between heavy analgesic ingestion and findings of SICK in incident US patients with ESRD, which seemed to be accounted for mostly by phenacetin-containing products; however, future studies are needed to learn whether there is an association in the US population between currently available analgesics and ESRD and whether the ingestion of NSAID or cyclooxygenase-2 inhibitors contribute to the development of ESRD.

Gastroparesis, a syndrome in which patients experience delayed gastric emptying, is characterized by symptoms such as nausea, vomiting, bloating, abdominal pain, and early satiety. Management of gastroparesis is limited by few effective treatments, many of which function by accelerating gastric emptying. Based on the hypothesis that some symptoms of gastroparesis arise because of neuropathic changes in enteric and sensory nerves, tricyclic antidepressants (TCAs) in low doses have been used as neuromodulators to treat refractory symptoms of nausea, vomiting, and abdominal pain, but there is little evidence to support this use. The Nortriptyline for Idiopathic Gastroparesis (NORIG) study is a multicenter, randomized, clinical trial that was designed by the Gastroparesis Clinical Research Consortium (GpCRC) to test whether treatment with nortriptyline, a TCA with reduced anticholinergic side effects, results in symptomatic improvement in patients with idiopathic gastroparesis. 

Individuals between the ages of 21 and 68 years old with moderate to severe symptoms of idiopathic gastroparesis for at least 6 months were enrolled. Participants were randomized to treatment with either nortiptyline or placebo. In both groups, dosing was escalated at 3-week intervals (10, 25, 50, 75 mg) up to 75 mg at 12 weeks. At follow-up study visits, which occurred every 3 weeks, symptom questionnaires were administered to assess gastrointestinal and psychological symptoms, quality of life, and TCA side effects. Electrogastrography (EGG) satiety tests and electrocardiography tests were also performed. Treatment was continued for 15 weeks, at which time study medication dose was tapered to zero with a final assessment at 18 weeks.  The primary outcome measure was a decrease from the patient’s baseline Gastroparesis Cardinal Symptom Index (GCSI) score of at least 50% on two consecutive 3 week GCSI assessments over 15 weeks of treatment. Secondary outcome measures included physiological assessments during satiety testing, clinical and psychological symptom scores, and adverse event rates.

Overall symptomatic improvement, as defined by the primary outcome measure, did not differ between the treatment groups: 23% on nortriptyline versus 21% on placebo. Additionally, treatment with nortriptyline showed no improvement in nausea, fullness/satiety, bloating, or quality of life measures. These findings suggest that TCAs may not be effective in the treatment of idiopathic gastroparesis.

The Biliary Atresia Research Consortium (BARC) is an NIH-funded network of 9 clinical sites and a data coordinating center whose goal is to study biliary atresia, neonatal hepatitis and other cholestatic diseases in the infant. BARC is preparing a prospective observational study of infants with cholestatic disease and clinical trials related to modalities of treatment.

Biliary atresia is the leading cause of morbidity and mortality from pediatric liver disease, and is the most common indication for pediatric liver transplantation. Despite its clinical importance, little is known of a wide range of fundamental issues regarding biliary atresia and optimal approaches to the medical management are unknown. No prospective randomized trials of important aspects of the care of children with biliary atresia have been performed. The majority of published data regarding biliary atresia is anecdotal and based upon a single center experience. Comprehensive registries of the management and outcome of biliary atresia do not exist.

This retrospective study will be unique and powerful because it analyzes the results of a large cohort of children in the United States from a diverse group of institutions with expertise in the management of children with liver disease. The proposed retrospective study may provide novel information and may lead to insights that can be used in the design of a subsequent 5 year prospective study.

Infants with cholestasis prior to 181 days of age present a challenging diagnostic problem of differentiation between an extra- and intra-hepatic location of the cholestatic process. This assessment is critical, since any evidence of obstruction essentially mandates surgical exploration. Extrahepatic biliary atresia (EHBA) is the most frequent cause of persistent unrelieved infant jaundice amenable to surgical correction.

This is a retrospective multi-center pathology project, which will examine interobserver variability in the histological diagnosis of infantile cholestasis with the aim of establishing reliable histological criteria of extra-hepatic obstruction in infants.

The CIT consortium conducted a total of 9 studies across North America (CIT02 through CIT08) and the Nordic region (CIT01). CIT08 was a long-term follow-up study for interested participants at the North American sites. The target population is individuals with type 1 diabetes, normal kidney function, and intractable hypoglycemia. All studies treated participants with up to 3 separate infusions of islets.

Non-Study Specific CIT data are available for request

Treatment of chronic hepatitis C in adults has evolved from alpha interferon (IFNα) alone to the combination of PEG-IFNα with ribavirin (RV). Recommendations for treatment for HCV infection in children have been derived from trials in adults, although the efficacy and safety of these therapies may be different in children. The Pediatric Study of Hepatitis C (PEDS-C) was conducted as a randomized, controlled trial to investigate the safety and efficacy of PEG-IFNα with and without RV in children and adolescents with chronic hepatitis C.  The primary objective of the PEDS-C study was to determine if the addition of RV to PEG-IFNα maximized outcome of therapy of children with chronic hepatitis C. 

Children and adolescents ages 5 to 18 years with documented chronic HCV infection were enrolled in the study. All participants were randomly assigned in a 1:1 ratio to receive either PEG-IFNα and RV or PEG-IFNα and placebo. PEG-IFNα was administered in a dose of 180 µg/1.73m2 body surface area subcutaneously once weekly to all subjects, and ribavirin was administered in a dose of 15 mg/kg orally twice daily to the appropriate treatment group. Patients without detectable HCV RNA at 24 weeks were continued on treatment for another 24 weeks, while those with detectable HCV RNA at 24 weeks were considered treatment failures.  The primary outcome measure was the proportion of subjects with a sustained virologic response, defined as non-detectable HCV RNA in plasma (<10 IU/ml) at least 24 weeks after stopping treatment. The secondary outcome measure was safety, assessed by vital signs, laboratory tests, and adverse events.

Results showed that therapy with PEG-IFNα plus RV was superior to PEG-IFNα plus placebo regardless of age, alanine aminotransferase (ALT) levels, and histologic severity. Additionally, there was little change in side effect profile with the addition of RV to PEG-IFNα. The study concluded that the combination of PEG-IFNα and RV is superior to PEG-IFNα alone as therapy for chronic hepatitis C in children and adolescents in both safety and efficacy.

Nonalcoholic steatohepatitis (NASH) is a common liver disease associated with obesity and diabetes. NASH is a progressive disorder that can lead to cirrhosis and liver failure, and there is no definitive treatment. Insulin resistance and oxidative stress (resulting in lipid peroxidation) are considered to be the two most important mechanisms in the pathogenesis of NASH.<br><br>PIVENS hypothesized that pioglitazone and vitamin E will lead to improvement in hepatic histology in nondiabetic adults with biopsy proven NASH through changes in insulin resistance or oxidative stress. Before and post-treatment liver biopsies were read centrally in a masked fashion for an assessment of steatohepatitis and a NAFLD Activity Score (NAS) consisting of steatosis, lobular inflammation, and hepatocyte ballooning.<br><br>PIVENS enrollment started in January 2005 and ended in January 2007 with 247 patients randomized to receive either pioglitazone (30 mg q.d.), vitamin E (800 IU q.d.), or placebo for 96 weeks. Participants were followed for an additional 24 weeks after stopping the treatment. Both at the initial evaluation and at the completion of treatment 96 weeks later, subjects underwent an assessment of body weight, height, and waist and hip circumferences, and blood samples were obtained for routine biochemical tests and assessment of fasting levels of lipids, glucose, and insulin. Body composition was assessed with the use of dual-energy x-ray absorptiometry. The Medical Outcomes Study 36-Item Short-Form Health Survey (SF-36) was administered for the assessment of quality of life. 

The complete PIVENS data is now available.

Epidemiological studies suggest that obesity is a strong risk factor for urinary incontinence (UI), and preliminary studies have suggested that weight loss may have a beneficial effect on urinary incontinence in obese women. The Program to Reduce Incontinence by Diet and Exercise (PRIDE) study was a multi-center (2 sites) randomized clinical trial conducted to examine the long term effects of a weight loss intervention on urinary incontinence in overweight and obese women. 

A total of 338 women with at least UI episodes per week and a body-mass index (BMI) between 25 and 50 were enrolled in the study. Participants were randomly assigned to an intensive 6-month behavioral weight-loss program (intervention group) or to a structured four-session education program (control group) and followed for 18 months. Following completion of the 6-month weight loss intervention, participants in the intervention group underwent a second randomization to a motivationally focused weight loss maintenance program or a standard skills-based maintenance approach. The secondary randomization allowed the comparison of two approaches to weight loss maintenance. Urinary incontinence was assessed with a 7-day voiding diary. Assessments were completed at 6, 12, and 18 months, and information was collected on height, weight, demographics, and medical history, including urinary  incontinence history. The primary outcome measure was the percentage change in the number of urinary incontinence episodes reported in the 7-day voiding diary at 6 months after randomization. Secondary outcome measures included the change in number of episodes of urge and stress urinary incontinence, participant satisfaction with incontinence treatment, and a standardized pad test to measure 24-hour involuntary urine loss. Longer term effects of the behavioral weight-loss program on UI were assessed at 12 and 18 months. 

Results showed that the comprehensive behavioral weight-loss program resulted in a significantly greater reduction in the frequency of self-reported UI episodes (mean weekly number of incontinence episodes) at 6 months as compared with the structured education program. Additionally, decreased stress incontinence episode frequency was maintained at 12 months with a trend toward continued reduction in total incontinence episode frequency. At 18 months, there were no statistically significant differences in the reduction of weekly incontinence episode frequency for stress or total urinary incontinence between the groups, despite a substantial difference in greater weight loss and participant satisfaction with improvements in UI in the intervention group. The study concluded that the behavioral weight loss intervention was more effective than an educational control program in decreasing stress-related UI through 12 months, although the benefits at 18 months were more limited.

This is a multi-center project to establish a prospective database of clinical information and a repository of blood and tissue samples from children with diagnoses of neonatal liver diseases, such as biliary atresia and neonatal hepatitis in order to perform research in these liver problems. Children were screened and enrolled at presentation at the participating pediatric liver sites. Participants diagnosed with biliary atresia were followed intensively for the first year, at 18 months of age, then annually up to 20 years of age, liver transplantation, or resolution of the underlying liver disease. Other subjects diagnosed with cholestasis were followed on the same schedule; if there was complete (clinical and biochemical) resolution of their underlying liver disease off all therapy, there was one follow up visit within one year (preferably scheduled at the time of the next planned follow up visit or at 12 months of age, whichever was later) for data collection and to obtain blood samples. The development of a serum and tissue bank of specimens from children with various neonatal cholestatic disorders are for use by future investigations into the etiology and pathogenesis of hepatobiliary injury in the infant.

The data package available for request includes analysis datasets from multiple publications including combined data from the <a href="https://repository.niddk.nih.gov/studies/basic/">BASIC</a>, <a href="https://repository.niddk.nih.gov/studies/logic/">LOGIC</a>, and <a href="https://repository.niddk.nih.gov/studies/start/">START</a> studies.

The RAND Interstitial Cystitis Epidemiology Study (RICE) is an observational study that sought to develop a case definition for Bladder Pain Syndrome (BPS)/Interstitial Cystitis (IC) with known sensitivity and specificity and to estimate the prevalence of the condition, which at the time had never been done using a large-population model. BPS/IC, which is clinically diagnosed based on patient-reported symptoms, is a poorly understood condition that can cause significant disability.

 An expert panel developed a case definitions of BPS/IC based on a review of medical literature. This definition was tested on 599 adult women diagnosed with BPS/IC or other conditions characterized by pelvic symptoms. Testing revealed that no single epidemiological definition demonstrated both high sensitivity and high specificity, so two definitions were developed: one of high sensitivity/low specificity and one of low sensitivity/high specificity.
Using these two definitions, researchers then screened 96,995 US households to identify women who had bladder symptoms. Those with symptoms underwent a secondary, more intensive screening and were asked to provide more information regarding symptoms, medical history, medical treatments, impact on daily life, physical and mental health, and demographic information.  Multivariate modeling was used to construct population and non-response weights. Weighted data was used to calculate prevalence estimates for both definitions and estimate the number of women affected.

Approximately 10 percent of Americans have diabetes and at least 33 percent of American adults are considered prediabetic.  The increasing loss of beta-cell function is one of the key factors in the pathogenesis of type 2 diabetes.  Over time, increased insulin resistance and beta-cell dysfunction can lead to serious health complications in diabetics.  Research efforts have begun to focus on the effects of certain interventions on insulin resistance and beta-cell function at the early stages of the disease.  The Restoring Insulin Secretion (RISE) Adult Medication Study was established to determine if pharmaceutical treatment could preserve or improve beta-cell function in adults with prediabetes or recent onset type 2 diabetes.

The RISE Adult Medication Study was a partially double-blind, placebo-controlled, four-arm clinical trial.  Study participants were randomized into four groups and received treatment for 12 months.  During the treatment period, the first group received Metformin alone; the second group received Glargine for three months, followed by Metformin for the remaining nine months; the third group received Liraglutide in combination with Metformin; and the fourth group was treated with placebo.  Following the 12-month treatment period, each group underwent treatment withdrawal for three months.  Insulin sensitivity and beta-cell function was evaluated at baseline, the end of the treatment period, and the end of the withdrawal period.

Obesity is one of the key risk factors in the development of type 2 diabetes.  Research has shown that excess body mass is linked to insulin resistance and loss of beta-cell function, which are precursors of type 2 diabetes. Clinical studies indicate medication and weight loss attained through lifestyle changes are both effective at slowing the progression of the disease by mitigating the effects of obesity on beta-cell function. The Beta Cell Restoration Through Fat Mitigation (BetaFat) Study was created to determine if weight loss through gastric banding surgery would be more effective than metformin medication at sustaining or improving beta-cell function in individuals with prediabetes or early type 2 diabetes, who are also mildly to moderately obese. 

BetaFat, a component of the Restoring Insulin Secretion (RISE) Consortium, was a two-arm, unblinded clinical trial. Study participants were recruited at the University of Southern California and randomized into two intervention groups.  One group received gastric banding surgery and the other group was treated with metformin. Patients underwent assessments at baseline and at predetermined intervals during the 24-month study period. Beta-cell function, insulin sensitivity, and blood glucose levels were regularly evaluated during the study period. Participants were also monitored for adverse effects to the interventions.

Although type 2 diabetes is more common in adults, there has been an increase in the number of children with the disease, primarily due to the obesity epidemic. As with adults, the decline of beta-cell function and insulin sensitivity are precursors of type 2 diabetes in children. There are several treatments for adults with type 2 diabetes, however insulin and metformin are the only approved treatments for children with type 2 diabetes. The purpose of the Restoring Insulin Secretion (RISE) Pediatric Medication Study was to determine whether metformin or insulin combined with metformin could be used to sustain or improve beta-cell function in children with prediabetes or recently diagnosed type 2 diabetes.

The RISE Pediatric Medication Study was a two-arm, multi-center clinical trial.  Study participants were enrolled at four recruitment centers and randomized into two treatment groups. The first group received metformin alone for 12 months. The second group received glargine, a man-made insulin, for three months followed by metformin for the remaining nine months of the treatment period. Beta-cell function and insulin sensitivity were assessed at baseline, the end of the active treatment period, and three months after treatment was withdrawn.

This multicenter, randomized, double-blind, placebo-controlled trial was designed to determine whether daily antimicrobial prophylaxis is superior to placebo in preventing recurrence of urinary tract infection (UTI) in children with vesicoureteral reflux (VUR). Patients were randomly assigned to treatment for 2 years with daily antimicrobial prophylaxis (trimethoprim-sulfamethoxazole) or placebo. The study was designed to recruit 600 children (approximately 300 in each treatment group. The protocol encouraged prompt evaluation of children with UTI symptoms and early therapy of culture-proven UTIs. It was expected that approximately 10% of children would have to discontinue study medication due to allergic reactions. Assuming a 20% placebo event rate and 10% non-adherence rate, the study had 83% power to detect an absolute 10% event rate in the antimicrobial prophylaxis group. If the placebo event rate was instead 25%, power was 97% to detect an absolute 10% event rate in the treated group, even if non-adherence were as high as 15%. The primary analysis is intention-to-treat with missing outcome data analyzed as UTI.

Characteristics associated with the phenotype of frailty include fatigue and reduced physiologic capacity in neurologic control, mechanical performance, and energy metabolism. Studies have shown that there is an increased risk of frailty in chronic disease populations, including patients with chronic kidney disease undergoing hemodialysis. Currently, little is known about the prevalence or the contributing factors of frailty among patients with this condition. The ACTIVE/ADIPOSE (a Cohort Study to Investigate the Value of Exercise in ESRD/Analyses Designed to Investigate the Paradox of Obesity and Survival in ESRD) study is a prospective, multicenter observational study designed to characterize the prevalence and progression of frailty among patients undergoing hemodialysis for end-stage renal disease (ESRD). 

Patients at least 18 years of age who were on hemodialysis for at least three months were enrolled in the study. Participants were assessed using the validated Fried frailty index, which defines frailty as the presence of 3 or more criteria, including recent unintentional weight loss, reported exhaustion, low grip strength, slow walk speed, and low physical activity. Information was collected on participants’ demographics, medical history, activity status, and mood, and physical performance was assessed via tests of grip strength, walk speed, and body composition. Additionally, blood draws were conducted semi-annually for evaluation of nutrition, inflammation, and cardiac markers. All participants were followed for two years. Logistic regression models were used to estimate the association of nutrition, cardiovascular, and rehabilitation parameters with patient frailty. 

Analyses showed that peripheral vascular disease and cardiac diseases, such as dysrhythmia, atrial fibrillation, tachycardia, pericarditis, and cardiac arrest, were associated with higher odds for frailty. In contrast, black race and higher serum albumin concentration were associated with lower odds for frailty. Among patients who met the Fried definition of frailty, 78% scored as frail on walk speed and 56% scored as frail on grip strength, the two measures of physical performance. The association of these sociodemographic and clinical risk factors with wasting in this cohort provides evidence for the prevalence of frailty in hemodialysis patients.

In response to a lack of basic descriptive epidemiology of urologic symptoms in the general populationthe Boston Area Community Health (BACH) Survey was established by the NIDDK to survey residents in the Boston metropolitan area about their urologic symptoms and how those symptoms affect their daily lives. The BACH Survey was designed to estimate the prevalence of urologic symptoms typical of  interstitial cystitis (IC), urinary incontinence, benign prostatic hyperplasia, prostatitis, hypogonadism, and impaired sexual function by race/ethnicity, age, sex, and socioeconomic status (SES). The survey created a random community-based sample of racially and ethnically diverse men and women across a broad age range, between 30 and 79 years.  The cohort consisted of 5502 participants, approximately equally divided between African American, Hispanic, and Caucasian individuals.   A substantial proportion of the originally recruited cohort was followed-up.

The investigators conducted a two-hour, in-home, bilingual field interview of all eligible participants, in which they examined symptoms and asked questions about lifestyle, physical activity, alcohol use, nutrition, demographics, and morbidity. They also conducted a detailed inventory of medications, both prescribed and over-the-counter, and took two non-fasting blood samples for hormone, cholesterol, and lipid levels. While the initial BACH study focused on urologic symptoms and conditions as its main outcomes of interest, two follow-up and a number of sub-studies were subsequently initiated on the BACH cohort with a focus on other health conditions in addition to urologic symptoms.

The BACH study contributed greatly to knowledge of the prevalence of urologic conditions, which was shown to be greater than previously estimated. The study succeeded in creating a large, racially-diverse sample to provide cross sectional, longitudinal data to explore urologic questions. This data has been used by researchers to identify gender, racial/ethnic, and socioeconomic disparities in a number of health conditions and is available for future studies.

The BARC study was a retrospective study that analyzed the results of a large cohort of patients with biliary atresia from US institutions with expertise in management of children with liver disease. The study consisted of a comprehensive retrospective chart review of clinical data and outcomes for all patients with biliary atresia undergoing HPE between January 1, 1997 and December 31, 2000 at 9 clinical centers. All children included in the study were followed for 2 years or until loss of the native liver as a result of transplantation or death. The data showed that there was no standard approach to the diagnosis of biliary atresia and that postsurgical management varied considerably among the BARC centers; nevertheless, the outcomes at age 2 years for the 9 centers were comparable to the best outcome data published from other countries and in large single-center reports. The study also showed that total bilirubin in early follow-up after HPE was highly predictive of outcome.

Data from the P001 and P002 protocols are now available.

The Chronic Prostatitis Collaborative Research Network (CPCRN) was established to conduct randomized controlled clinical trials of promising therapies for chronic prostatitis/chronic pelvic pain syndrome (CP/CPPS) in order to treatment of this syndrome. While the pathogenesis of chronic prostatitis/chronic pelvic pain syndrome (CP/CPPS) is not known, there is some evidence to suggest that the urogenital pain of CP/CPPS may be neuropathic. The CPCRN conducted a randomized clinical trial to investigate whether a drug found to be effective for treating neuropathic pain, pregabalin, could be beneficial in treating the symptoms of CP/CPPS. 

Men with CP/CPPS form 10 clinical centers were enrolled and randomly assigned 2:1 in each clinical site to receive treatment with either pregabalin or matching placebo (the trial was double-blind). Treatment was for six weeks.  Dose was escalated from 150 mg/d (50 mg orally 3 times daily) for 2 weeks, to 300 mg/d (100 mg orally 3 times daily) for 2 weeks, and then to 600 mg/d (200 mg orally 3 times daily) for 2 weeks; participants in the placebo group underwent a similar dose escalation. The primary outcome measure was a decrease (improvement) in the National Institutes of Health Chronic Prostatitis Symptom Index (NIH-CPSI) score of at least 6 points from baseline to week 6. Several secondary outcome measures were assessed, including the subscores of the NIH-CPSI, the Global Response Assessment (a self-reported assessment that measures perception of change in symptoms), the Medical Outcomes Study 12-Item Short Form Health Survey, the Hospital Anxiety and Depression Scale, and the Sexual Health Inventory for Men. 

The study found no a clinically significant (6-point) decrease in the NIH-CPSI in men treated with pregabalin compared to those assigned to placebo.

The CyNCh (Cysteamine Bitartrate Delayed-Release for the Treatment of NAFLD in Children) study is a prospective, multicenter, double-blind clinical trial designed by the NASH Clinical Research Network to determine whether treatment with cysteamine improves disease severity in children diagnosed with NAFLD.  Children between the ages of 8 and 17 years with biopsy-confirmed moderate to severe NAFLD were eligible for the CyNCh study. Participants were enrolled and randomized to treatment with either delayed-release cysteamine bitartrate capsules or placebo capsules. Improvement in NAFLD at 52 weeks, defined as a decrease in NAFLD Activity Score (NAS) of at least 2 and no worsening of fibrosis, was assessed as the primary outcome measure. Secondary outcome measures, assessed at 52 weeks, included reduction in serum aminotransferase and gamma-glytamyl transpeptidase, reduction in MRI-determined hepatic fat fraction, changes in markers of oxidation and anti-oxidant status, changes in histology and symptoms, and self-reported quality of life.

2Acute kidney injury is a common complication of acute illness, affecting approximately 2 to 7% of hospitalized patients and more than 35% of critically ill patients. While renal-replacement therapy is the main treatment method for patients with acute kidney injury, the optimal timing for the initiation, method, and dosing of therapy is uncertain. Given this uncertainty, the Acute Renal Failure Trial Network (ATN) study was established to determine whether more intensive renal replacement therapy decreases mortality among critically ill patients with acute kidney injury. 

The study was a multicenter, prospective, randomized trial of two strategies for renal-replacement therapy in patients with acute kidney injury. Participants were randomized to receive intensive (i.e., intermittent hemodialysis and sustained low-efficiency dialysis six times per week and continuous venovenous hemodiafiltration at 35 ml/kg of body weight/hour) or less intensive renal-replacement therapy (i.e., the corresponding treatments were provided three times per week and at 20 ml/kg/hour). The assigned renal-replacement therapy was provided for up to 28 days after randomization or until recovery of kidney function, discharge from acute care, withdrawal of life-sustaining therapy, or death. The primary outcome measure was death from any cause by day 60. Recovery of kidney function (defined as lack of need for continuing dialysis support, with a minimum creatinine clearance of 20 ml/minute), duration of renal-replacement therapy, lengths of stay in the intensive care unit (ICU) and hospital, and days free of nonrenal organ failure were also assessed. 

Results showed that the primary outcome measure, death from any cause by day 60, did not differ between the two groups. Additionally, recovery of renal function and reduction in the rate of nonrenal organ showed no improvement with intensive renal support. The study found no added benefit from an intensive (high-dose) treatment strategy as compared with the more conventional, less-intensive treatment strategy.

The Diabetes Prevention Trial—Type 1 Diabetes (DPT-1) consisted of two randomized clinical trials to determine whether insulin could prevent or delay the onset of overt diabetes in relatives of patients with diabetes. One study investigated the effects of parenteral insulin in relatives with a projected five-year risk of diabetes higher than 50 percent, while the other looked at the effects of oral insulin in relatives with a projected five-year risk of 26 to 50 percent.
 
First and second-degree relatives were screened for islet-cell antibody (ICA) titer ≥ 10 and then staged to assess risk of diabetes. Those with a high risk were eligible for the parenteral insulin trial, and those with a low risk were eligible for the oral insulin trial. In the parenteral insulin trial, subjects in the intervention group received parenteral insulin subcutaneous injections twice daily, plus annual intravenous infusions; the control group received no treatment. In the oral insulin trial, subjects were assigned to receive capsules of either oral insulin or matched placebo. For both trials, participants were seen every 6 months, and at those visits an oral glucose tolerance (OGT) test was performed to assess glycemic status, the primary study end point. Other assessments included intravenous glucose tolerance tests and mixed-meal tolerance tests performed at baseline, as well as at various points during the study. Results showed that neither parenteral nor oral insulin succeeded in delaying or preventing the development of diabetes in high-risk relatives of current diabetes patients. Long-term follow-up to detect any effects on the course of diabetes was initiated.

The Clinical Outcomes Research Initiative (CORI) was established in 1995 under the American Society for Gastrointestinal Endoscopy (ASGE) to study outcomes of gastrointestinal (GI) endoscopic procedures in clinical settings. Physicians participating in the CORI consortium produce GI endoscopy reports using an electronic health record developed specially for the project. CORI practice sites include hospitals, ambulatory care centers, private practices, universities, and Veteran's hospitals. The practice data are stripped of most patient and physician identifiers before transmitting to a central data repository, where they are tested for completeness and accuracy. Data from all participating practices are merged and stored in the National Endoscopic Database (NED).

Data from the NED has been analyzed to examine endoscopic practice patterns, including endoscopic utilization, frequency and severity of endoscopic findings, and endoscopic treatment and medical management. The data also serve as a resource to develop research hypotheses and to support quality measure reporting.  CORI data has already been utilized to support many research initiatives, many of which have resulted in publications in medical journals and presentations at GI conferences. 

In addition to availability in through the CORI consortium, the data collected in the NED since 2000 is been contributed to the NIDDK Repository and is available for request through the NIDDK Repository site. 

The current data package contains data from the v3 warehouse from 2000 through 2010.

The second phase of the Chronic Prostatitis Collaborative Research Network (CPCRN chronic prostatitis/chronic pelvic pain syndrome (CP/CPPS) was established to conducted randomized clinical trials of promising therapies for this syndrome. In men with CP/CPPS , treatment with alpha-adrenergic receptor blockers early in the course of the disorder has been reported to be effective in some, but not all, relatively small randomized trials. In response to the findings of these previous trials, the CPCRN conducted a multicenter, randomized, placebo-controlled trial of alfuzosin to determine whether the symptoms CP/CPPS could be reduced in men who had recently received a diagnosis of CP/CPPS and who had not previously been treated with this class of drug.

Men with CP/CPPS were enrolled at 11 recruitment centers and randomly assigned in a 1:1 ratio to receive either 10 mg of alfuzosin or placebo once daily for 12 weeks. The trial was double-blind.  There were four research-clinic visits during which data for the primary and secondary outcome measures were collected. The primary outcome measure was a decrease (improvement) in the National Institutes of Health Chronic Prostatitis Symptom Index (NIH-CPSI) of at least 4 points from baseline to 12 weeks, which is the minimal clinically significant difference in the score. Secondary outcome measures included evaluations of pain, urinary urgency, medical outcomes, mental health, and erectile dysfunction. Adverse events associated with treatment was assessed for each subject both overall and within each body system. 

Results showed no difference between the placebo and the alfuzosin groups in the proportion of men with in an improvement of the NIH-CPSI score and a clinically meaningful reduction of symptoms. Similarly, there was no significant difference between the alfuzosin and placebo groups in multiple secondary outcomes, including the results of the global response assessment and measures of quality of life, depression, sexual function, and pain. These findings do not support the use of alfuzosin to reduce the symptoms of CP/CPPS in men who have not received prior treatment with an alpha-blocker.

Autosomal dominant polycystic kidney disease (ADPKD) impacts 1 in 400 to 1 in 1000 live births and accounts for about 4.6% of the kidney replacement population in the US. Hypertension is the most common manifestation and an important risk factor for its progression to end stage renal disease (ESRD) and cardiovascular morbidity and mortality.

The HALT Progression of Polycystic Kidney Disease (HALT PKD) studies were two simultaneous multicenter clinical trials designed to test the efficacy of interruption of the renin-angiotensin-aldosterone system (RAAS) on the progression of cystic disease and the decline in renal function in ADPKD. Specifically, the studies assessed the effects of ACE-I/ARB combination therapy as compared to ACE-I monotherapy in hypertensive ADPKD subjects.

The two studies varied on eligibility criteria (particularly kidney function), blood pressure treatment, and primary outcome measure. Study A investigates treatment effects on individuals with early ADPKD and implements a standard (120-130/70-80 mm Hg) vs. low (95-110/60-75 mmHg) blood pressure control target for both treatment arms in a 2x2 design. Study B investigates treatment effects on individuals with moderately advanced ADPKD in the setting of standard blood pressure control (110-130/80 mm Hg). Participants enrolled in study A were followed for at least five years, while those enrolled in study B were followed for five to eight years, with the average length of follow-up being six and a half years.

The complete HALT PKD data package is available for request.

MRI images for HALT PKD participants are not included in the package, but are available upon request.

The Hemodialysis (HEMO) Study was a multi-center, randomized clinical trial designed to test whether the mortality and morbidity of hemodialysis (HD) patients can be reduced by modifying the dialysis procedure for the removal of toxins from the blood. The study examined two factors, the dose of dialysis delivered and the size of the molecules removed, to determine the effect on the survival or morbidity among hemodialysis patients. A two-by-two factorial design was used to assign 1846 patients randomly to either a standard or high dose of dialysis and to either a dialyzer with a low-flux or high-flux membrane for dialysis three times a week. The primary outcome, death from any cause, was not significantly influenced by the dose or flux assignment. The relative risk of death was not significantly different in the high-dose group compared to the standard-dose group, or the high-flux group as compared with the low-flux group. The main secondary outcomes, including first hospitalization, did not differ significantly for either the dose groups or the flux groups. Patients undergoing hemodialysis three times a week appear to have no major benefit from either a higher dialysis dose than that recommended by current U.S. guidelines or from the use of a high-flux membrane.

Irritable bowel syndrome (IBS) is a condition which affects the large intestine, with symptoms that include varying degrees of abdominal pain, bloating, constipation and diarrhea.  Medications and lifestyle changes are generally prescribed for IBS, but medical treatments for the full range of symptoms are inadequate.  Cognitive behavior therapy (CBT), a psychological treatment where therapists teach patients how to use behavioral skills to manage IBS and relieve unresolved symptoms, has been associated with improvements in symptom severity and quality of life in other clinical trials.  However, CBT has certain economic and feasibility limitations that prevent the therapy from being incorporated into routine clinical settings.

The Irritable Bowel Syndrome Outcome Study (IBSOS) sought to address the limitations of standard therapist-administered CBT by developing an effective self-administered version of the treatment method.  IBSOS participants were randomized into one of three treatment groups and underwent four weeks of symptom monitoring.  One group received the standard ten sessions of clinic-based CBT, one group received four sessions of minimal-contact CBT, and the remaining group received four sessions of supportive counseling and education without any prescribed behavior changes.  Following the symptom monitoring phase, patients underwent treatment and follow-up phases.  Assessments were regularly conducted during the study and the results were evaluated between the groups.

The Interstitial Cystitis Data Base (ICDB) cohort study was a prospective, multicenter clinical study of patients with Interstitial Cystitis (IC). The study gathered data to investigate the treated history of IC and identify patient subgroups with specific symptoms, physical findings, histopathologic findings from bladder biopsies, and treatment responses. Data was collected on patients at baseline and longitudinally concerning demographic and diagnostic information, medical history, dietary intake, urinary symptoms, and treatments and their outcomes. Additionally, bladder tissue specimens were stored in a specimen bank.  Initial analyses on patient data supported the clinical observation that IC is a chronic symptom complex and showed that no current treatments had a significant impact on symptoms over time.

Short-term studies have shown numerous benefits of weight loss in overweight or obese patients, including improvements in glycemic control, risk factors for cardiovascular disease, quality of life, and other obesity-related coexisting illnesses. The Look AHEAD study was designed to test whether weight loss similarly improved cardiovascular morbidity and mortality in patients with type 2 diabetes. The study is a multicenter, randomized clinical trial that examines the long-term effects of an intensive lifestyle intervention program designed to achieve and maintain weight loss by decreased caloric intake and increased physical activity.

Eligible patients with type 2 diabetes and a body-mass index (BMI) of 25.0 or more were enrolled and randomly assigned either to participate in an intensive lifestyle intervention (intervention group) or to receive diabetes support and education (control group). The intensive lifestyle intervention, which included both group and individual counseling sessions, was aimed at achieving and maintaining weight loss of at least 7% by focusing on reduced caloric intake and increased physical activity. The diabetes support and education program featured sessions focusing on diet, exercise, and social support. Both the intervention and control programs occurred with decreasing frequency as the trial progressed. The primary outcome measure is the first occurrence of a composite cardiovascular outcome, which consists of death from cardiovascular causes, nonfatal myocardial infarction, nonfatal stroke, and hospitalization for angina. Participants will be followed for a planned period of 13.5 years.

An ancillary study, Look AHEAD Brain, was conducted to assess whether participation in the 10-year lifestyle intervention, as part of Look AHEAD, had an impact on white matter hyperintensity and loss of brain tissue among individuals with type 2 diabetes. A subset of Look AHEAD study participants underwent standardized brain magnetic resonance imaging in conjunction with tests assessing cognitive function 10-12 years post-randomization.

The Look AHEAD intensive lifestyle intervention ended in September, 2012. Participants continued to be followed to determine the long-term effects of the intervention on health outcomes.

The Look AHEAD Continuation Study (Look AHEAD-C) builds on the Look AHEAD study to determine the long-term impact of an intensive lifestyle intervention on 1) physical function and mobility disability, and 2) cognitive function and cognitive impairment. Collection of cognitive function measures began in Year 8 of the study and continued through Year 13. 

The current data package contains data through the end of the post intervention program.

The NIDDK Liver Transplantation Database was established to prospectively investigate questions related to the experience of patients evaluated for and undergoing liver transplantation. The LTD contains data for 1563 patients referred and evaluated for orthotopic liver transplantation (OLT) at three large liver transplant centers in the United States; of these patients, 916 underwent transplantation and were followed for 1 to 5 years. 

All patients referred for OLT were screened for eligibility to be entered in the database, which required informed consent, no prior liver transplant, and availability to be followed. For each participant, standardized data was collected on demographics, medical history, preoperative and post-transplant symptoms and quality of life, post-transplant complications, and mortality. Liver biopsies and serum samples were also obtained pre- and post-transplantation at intervals specified in the study protocol. 

Following primary data collection, which occurred from April 1990 to June 1994, the Liver Transplantation Database Follow Up (LTD2) study was conducted to prospectively generate detailed survival data from a large multicenter cohort of liver transplant recipients. This study observed the long-term outcomes of transplantation to examine adverse effects, assess risk factors, and determine long-term mortality rates and causes of death post-transplant. 

Follow-up data was obtained for 798 transplant patients from 1998 to 2003 at 3 clinical centers. Cause of death was captured in the early (≤ 1 year) and late (> 1 year and > 5 years) post-transplant periods. Patient-related risk factors were analyzed for overall deaths as well as for each major cause of death in the late post-transplant period. Adverse events, including complications of transplantation and recurrence or development of disease, were monitored. 

The follow-up study identified common etiologies and risk factors that contributed to the morality of liver transplant patients at various stages post-transplantation. Results showed that hepatic etiologies were the most frequent cause of death in the late post-transplant setting. In addition to malignancy and cardiovascular disease, renal failure was found to be an important and escalating cause of death in the late post-transplant setting. Ancillary studies have also been performed on LTD data to evaluate pathology reliability and cost of liver transplantation, as well as examine various characteristics or biomarkers of collected serum.

Lower urinary tract dysfunction (LUTD) is a condition related to the functions and/or structures of the lower urinary tract.  LUTD affects both men and women, and becomes more common and frequent with age.  Patients tend to exhibit a range of lower urinary tract symptoms (LUTS), but there has been a lack of understanding regarding which biological and psychosocial factors cause the symptoms to manifest and worsen.  As a result, treatment and management methods for LUTS and LUTD have been unsatisfactory.  

The Symptoms of Lower Urinary Tract Dysfunction Phenotyping Study (LURN) was established to gain a better understanding of lower urinary tract symptoms in an effort to improve the lives of those with LUTD.  LURN participants were enrolled at multiple sites within the United States.  Participants were assigned to one of four subgroups, based on their symptoms, the outcomes of clinical assessments, and other characteristics.  Biological samples were collected from study participants and participants were required to complete certain assessments and examinations at standard time-points during the study period.

MRI images for LURN participants are not included in the package, but are available upon request.

Overview

Timeline

Prize

Rules

Judging Criteria