The National Institute of Diabetes & Digestive & Kidney Disease

Liver Diseases Databases, Registries and Information

The Acute Liver Failure Study Group is collecting biosamples and information on the natural history, causes and outcomes of Acute Liver Failure in the United States. In addition to the database, a clinical trial conducted to test whether the drug N-acetylcysteine (NAC) improves outcome (survival) for patients with Acute Liver Failure not caused by acetaminophen overdose has recently been completed. Results should be available in the near future.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

On July 1, 2003, The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) of the National Institutes of Health (NIH) established Central NIDDK Repositories for biosamples and data collected in clinical studies. The purpose of the Central Repositories is to expand the usefulness of these studies by providing access to the biosamples and data to a wider research community beyond the end of the study.

For more information, contact Dr. Rebekah Rasooly, Deputy Director of the Division of Kidney, Urologic, and Hematologic Diseases

A collection of statistics about specific digestive diseases, including prevalence, mortality, care delivery and cost.

The Centers are housed at outstanding academic institutions, staffed by experts in state-of-the-art technology. Researchers can ship mice to one of the four Centers and obtain on a fee-for-service basis a range of complex exams used to characterize mouse metabolism, blood composition including hormones, energy balance, eating and exercise, organ function and morphology, physiology and histology. Many tests are done in living animals and are designed to elucidate subtle to complex traits that would define models of metabolic disease.

For more information, contact Dr. Maren Laughlin, DEM, Senior Advisor for Integrative Metabolism

The National Gene Vector Laboratories (NGVL) are composed of an interactive group of academic production and pharm/tox laboratories whose primary goal is to provide eligible investigators with clinical grade vectors for phase I/II gene therapy clinical trials and to provide support for relevant pharmacology/toxicology studies leading up to clinical gene transfer protocols. If the application is approved, clinical grade material will be produced at no cost to the investigator.

For more information, contact Dr. Catherine McKeon, DEM, Senior Advisor for Genetic Research in Diabetes, Endocrinology and Metabolic Diseases

The Nuclear Receptor Resource (NRR) Project is a collection of individual databases on members of the steroid and thyroid hormone receptor superfamily. Although the databases are located on different servers and are managed individually, they each form a node of the NRR. The NRR itself integrates the separate databases and allows an interactive forum for the dissemination of information about the superfamily.

For more information, contact Dr. Ronald Margolis, DEM, Senior Advisor, Molecular Endocrinology

Commensurate with this directive, NURSA's goals can be distilled into two broad aims: (i) to execute research strategies designed to rapidly and efficiently elucidate those facets of orphan nuclear receptor biology we deem most critical to its understanding; and (ii) to facilitate the generation of hypotheses, design of experiments and communication of results by scientists active in this field. We anticipate that this initiative will provide a valuable service to the nuclear receptor community by developing a web-accessible bioinformatics resource, in which current and emerging data will be organized into more accessible and "user-mineable" forms.

For more information, contact Dr. Ronald Margolis, DEM, Senior Advisor, Molecular Endocrinology

The U.S. Organ Procurement and Transplantation Network (OPTN) maintains a registry of human tissues in order to ensure the success and efficiency of the U.S. organ transplant system.

For more information, contact Dr. Thomas Eggerman, DEM, Director, Islet Transplantation Clinical Trials Program

This multi-center, multi-national collaborative group of pediatric clinical liver centers is aimed at identifying, characterizing, and developing management strategies for infants, children, and adolescents who present with acute liver failure (ALF). In addition to a database of pediatric patients with ALF, a clinical trial is being conducted to test whether the drug N-acetylcysteine (NAC) improves outcome (survival) for patients with ALF not caused by acetaminophen overdose.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

Liver Diseases Multicenter Clinical Research

The Acute Liver Failure Study Group is collecting biosamples and information on the natural history, causes and outcomes of Acute Liver Failure in the United States. In addition to the database, a clinical trial conducted to test whether the drug N-acetylcysteine (NAC) improves outcome (survival) for patients with Acute Liver Failure not caused by acetaminophen overdose has recently been completed. Results should be available in the near future.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

The primary goal of A2ALL is to provide valuable information on the outcomes of both potential and actual recipients and their donors who have been evaluated for adult-to-adult living donor liver transplantation.

For more information, contact Dr. Jay Everhart, DDN, Director, Epidemiology and Data Systems Branch

The Biliary Atresia Research Consortium is a network of ten pediatric liver centers across the United States whose aim is to study causes and improve the diagnosis and treatment of infants and children with neonatal hepatitis and biliary atresia. Studies include a prospective database of infants with cholestatis and biliary atresia; A randomized, double-blinded, placebo-controlled trial of corticosteroid therapy following portoenterostomy in infants with biliary atresia; and, a database of older children with biliary atresia.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

The Cholestatic Liver Disease Consortium (CLiC) is a network of ten pediatric liver centers, core research facilities and patient support organizations throughout the US and London, UK working together to investigate five genetic causes of intrahepatic cholestasis and to train the next generation of clinical researchers.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

The Clinical Trials in Organ Transplantation (CTOT) project is a cooperative research program sponsored by NIAID, NIDDK & NHLBI. This consortium is conducting clinical and associated mechanistic studies to facilitate improved outcomes for abdominal (kidney & liver) and thoracic (heart & lung) transplant recipients; short and long-term graft and patient survival.

For more information, contact Dr. Catherine Meyers, KUH, Director, Inflammatory Kidney Diseases Program

To promote research on the causes of drug-induced liver disease, the National Institute of Diabetes and Digestive and Kidney Diseases has created the Drug-Induced Liver Injury Network (DILIN). Its purpose is to collect and carefully analyze cases of liver problems caused by prescription drugs and alternative medicines, such as herbal products.

For more information, contact Dr. Jose Serrano, DDN, Director, Liver and Biliary Program and Pancreas Program

A consortium of 20 clinical sites and one data coordinating center studying the effect of antiviral therapy on long term outcome after liver transplantation for chronic hepatitis B virus infection. Enrollment is complete and analysis and ancillary studies are ongoing.

For more information, contact Dr. Edward Doo, DDN, Director, Liver Diseases Program

HALT-C is a multi-center randomized clinical trial to determine if four years of interferon therapy can prevent progression of liver disease in persons with severe liver injury who have not cleared hepatitis C virus with standard therapy.

For more information, contact Dr. Jay Everhart, DDN, Director, Epidemiology and Data Systems Branch

This is a multicenter, randomized, controlled trial of high dose ursodiol versus placebo for patients with primary sclerosing cholangitis (PSC). The average duration of follow-up will be approximately five years with important clinical endpoints such as death, eligibility for liver transplanation, changes in histology and cholangiogram as well as liver biochemistries and quality of life data collected. Enrollment is now complete.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

The Nonalcoholic Steatohepatitis Clinical Research Consortium (NASH CRN) is a collaborative group of eight adult and pediatric clinical research centers and a data coordinating center created to focus on the etiology, contributing factors, natural history, complications, and therapy of nonalcoholic steatohepatitis. A long term database and a two clinical trials, one in adults and one in children, are ongoing.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

This multi-center, multi-national collaborative group of pediatric clinical liver centers is aimed at identifying, characterizing, and developing management strategies for infants, children, and adolescents who present with acute liver failure (ALF). In addition to a database of pediatric patients with ALF, a clinical trial is being conducted to test whether the drug N-acetylcysteine (NAC) improves outcome (survival) for patients with ALF not caused by acetaminophen overdose.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

The main purpose of this study is to determine the safety and efficacy of pegylated interferon in combination with ribavirin alone for the treatment of chronic hepatitis C virus infection in children. Enrollment in this study is complete.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

Studies of Pediatric Liver Transplantation, SPLIT, is a research effort that was organized in 1995 by a group of physicians and surgeons committed to the success of pediatric liver transplantation. The group represents a cooperative effort between transplant centers in the United States and Canada to prospectively collect and analyze information required to advance the science of pediatric liver transplantation.

For more information, contact Dr. Edward Doo, DDN, Director, Liver Diseases Program

A study conducted at eight clinical sites and included ancillary studies at four additional sites to evaluate factors associated with resistance to antiviral therapy in 400 African American and Caucasian American patients with chronic hepatitis C. All patients were treated with combination therapy of pegylated interferon and ribavirin for 48 weeks, and were followed for an additional 48 weeks after cessation of therapy. The study has been completed. Analyses and ancillary studies are ongoing.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

Liver Diseases Basic Research Networks

On July 1, 2003, The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) of the National Institutes of Health (NIH) established Central NIDDK Repositories for biosamples and data collected in clinical studies. The purpose of the Central Repositories is to expand the usefulness of these studies by providing access to the biosamples and data to a wider research community beyond the end of the study.

For more information, contact Dr. Rebekah Rasooly, Deputy Director of the Division of Kidney, Urologic, and Hematologic Diseases

To promote research on the causes of drug-induced liver disease, the National Institute of Diabetes and Digestive and Kidney Diseases has created the Drug-Induced Liver Injury Network (DILIN). Its purpose is to collect and carefully analyze cases of liver problems caused by prescription drugs and alternative medicines, such as herbal products.

For more information, contact Dr. Jose Serrano, DDN, Director, Liver and Biliary Program and Pancreas Program

The Centers are housed at outstanding academic institutions, staffed by experts in state-of-the-art technology. Researchers can ship mice to one of the four Centers and obtain on a fee-for-service basis a range of complex exams used to characterize mouse metabolism, blood composition including hormones, energy balance, eating and exercise, organ function and morphology, physiology and histology. Many tests are done in living animals and are designed to elucidate subtle to complex traits that would define models of metabolic disease.

For more information, contact Dr. Maren Laughlin, DEM, Senior Advisor for Integrative Metabolism

NMRI is a communication network of current and potential biomedical research investigators and technical personnel from traditionally under-served communities: African American, Hispanic American, American Indian, Alaskan Native, Native Hawaiian, and other Pacific Islanders. The major objective of the network is to encourage and facilitate participation of members of underrepresented racial and ethnic minority groups in the conduct of biomedical research in the fields of diabetes, endocrinology, metabolism, digestive diseases, nutrition, kidney, urologic and hematologic diseases. A second objective is to encourage and enhance the potential of the underrepresented minority investigators in choosing a biomedical research career in these fields. An important component of this network is promotion of two-way communications between network members and the NIDDK.

For more information, contact Ms. Winnie Martinez, Program Analyst, Office of Minority Health Research Coordination

Commensurate with this directive, NURSA's goals can be distilled into two broad aims: (i) to execute research strategies designed to rapidly and efficiently elucidate those facets of orphan nuclear receptor biology we deem most critical to its understanding; and (ii) to facilitate the generation of hypotheses, design of experiments and communication of results by scientists active in this field. We anticipate that this initiative will provide a valuable service to the nuclear receptor community by developing a web-accessible bioinformatics resource, in which current and emerging data will be organized into more accessible and "user-mineable" forms.

For more information, contact Dr. Ronald Margolis, DEM, Senior Advisor, Molecular Endocrinology

This multi-center, multi-national collaborative group of pediatric clinical liver centers is aimed at identifying, characterizing, and developing management strategies for infants, children, and adolescents who present with acute liver failure (ALF). In addition to a database of pediatric patients with ALF, a clinical trial is being conducted to test whether the drug N-acetylcysteine (NAC) improves outcome (survival) for patients with ALF not caused by acetaminophen overdose.

For more information, contact Dr. Patricia Robuck, DDN, Program Director for Clinical Trials in Digestive Diseases and Nutrition Program

Liver Diseases Reagents

On July 1, 2003, The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) of the National Institutes of Health (NIH) established Central NIDDK Repositories for biosamples and data collected in clinical studies. The purpose of the Central Repositories is to expand the usefulness of these studies by providing access to the biosamples and data to a wider research community beyond the end of the study.

For more information, contact Dr. Rebekah Rasooly, Deputy Director of the Division of Kidney, Urologic, and Hematologic Diseases

The National Gene Vector Laboratories (NGVL) are composed of an interactive group of academic production and pharm/tox laboratories whose primary goal is to provide eligible investigators with clinical grade vectors for phase I/II gene therapy clinical trials and to provide support for relevant pharmacology/toxicology studies leading up to clinical gene transfer protocols. If the application is approved, clinical grade material will be produced at no cost to the investigator.

For more information, contact Dr. Catherine McKeon, DEM, Senior Advisor for Genetic Research in Diabetes, Endocrinology and Metabolic Diseases

Liver Diseases Services

A centralized facility established to provide genotyping and statistical genetics services for investigators seeking to identify genes that contribute to human disease. CIDR concentrates primarily on multifactorial hereditary disease although linage analysis of single gene disorders can also be accommodated.

For more information, contact Dr. Catherine McKeon, DEM, Senior Advisor for Genetic Research in Diabetes, Endocrinology and Metabolic Diseases

On July 1, 2003, The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) of the National Institutes of Health (NIH) established Central NIDDK Repositories for biosamples and data collected in clinical studies. The purpose of the Central Repositories is to expand the usefulness of these studies by providing access to the biosamples and data to a wider research community beyond the end of the study.

For more information, contact Dr. Rebekah Rasooly, Deputy Director of the Division of Kidney, Urologic, and Hematologic Diseases

The Centers are housed at outstanding academic institutions, staffed by experts in state-of-the-art technology. Researchers can ship mice to one of the four Centers and obtain on a fee-for-service basis a range of complex exams used to characterize mouse metabolism, blood composition including hormones, energy balance, eating and exercise, organ function and morphology, physiology and histology. Many tests are done in living animals and are designed to elucidate subtle to complex traits that would define models of metabolic disease.

For more information, contact Dr. Maren Laughlin, DEM, Senior Advisor for Integrative Metabolism

NIH RAID provides a variety of contract services researchers need to bring promising potential therapeutics to trial.

Liver Diseases Standardization Programs

Liver Diseases Tissues, Cells, Animals

On July 1, 2003, The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) of the National Institutes of Health (NIH) established Central NIDDK Repositories for biosamples and data collected in clinical studies. The purpose of the Central Repositories is to expand the usefulness of these studies by providing access to the biosamples and data to a wider research community beyond the end of the study.

For more information, contact Dr. Rebekah Rasooly, Deputy Director of the Division of Kidney, Urologic, and Hematologic Diseases

The Liver Tissue Procurement and Distribution System (LTPADS) is a National Institutes of Health (NIH) service contract to provide human liver from regional centers for distribution to scientific investigators throughout the United States. These USA regional centers have active liver transplant programs with human subjects' approval to provide portions of the resected pathologic liver for which the transplant is performed. Frozen or fresh tissue is available from subcontractors for the usual forms of childhood and adult cirrhosis, fulminate liver failure, chronic rejection, and certain inborn errors of metabolism. “Normal” liver specimens may be requested, however, the supply is appropriately very limited and completion of large proposal requests is unlikely. A new service is now offered to provide isolated hepatocytes only to NIH investigators from "normal" human liver.

For more information, contact Dr. Jose Serrano, DDN, Director, Liver and Biliary Program and Pancreas Program

The Centers are housed at outstanding academic institutions, staffed by experts in state-of-the-art technology. Researchers can ship mice to one of the four Centers and obtain on a fee-for-service basis a range of complex exams used to characterize mouse metabolism, blood composition including hormones, energy balance, eating and exercise, organ function and morphology, physiology and histology. Many tests are done in living animals and are designed to elucidate subtle to complex traits that would define models of metabolic disease.

For more information, contact Dr. Maren Laughlin, DEM, Senior Advisor for Integrative Metabolism

The goal of the MMRRC program is to enhance the availability of and help ensure the quality of genetically modified mice for biomedical research of human and animal biology and disease.

For more information, contact Dr. Kristin Abraham, DEM, Director, Cell Signaling and Diabetes Centers Program

Liver Diseases Useful Tools