A Prospective Database of Infants With Cholestasis
- Conditions
- Biliary AtresiaNeonatal Cholestasis
- Registration Number
- NCT00061828
- Lead Sponsor
- Arbor Research Collaborative for Health
- Brief Summary
Biliary atresia, idiopathic neonatal hepatitis, and specific genetic cholestatic conditions are the most common causes of jaundice and hyperbilirubinemia that continue beyond the newborn period. The long term goal of the Childhood Liver Disease Research Network (ChiLDReN) is to establish a database of clinical information and plasma, serum, and tissue samples from cholestatic children to facilitate research and to perform clinical, epidemiological and therapeutic trials in these important pediatric liver diseases.
- Detailed Description
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 (BA), idiopathic neonatal hepatitis (INH), and specific neonatal presentations of genetic cholestatic disorders in order to perform research in these important liver problems. Children will be screened and enrolled at presentation at the participating pediatric liver sites. Participants diagnosed with BA will be followed intensively for the first year, at 18 months of age, and then annually up to 20 years of age, or liver transplantation. Other participants diagnosed with cholestasis will be followed on the same schedule; if there is complete (clinical and biochemical) resolution of their underlying liver disease off all therapy, there will be 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 is 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 will be an invaluable tool for current and future investigations into the etiology and pathogenesis of hepatobiliary injury in the infant.
Detailed clinical data, laboratory investigations, liver and biliary specimens, and long-term follow-up of outcomes are part of the normal standard of care with respect to the diagnosis and treatment of the subjects with liver problems. This research involves the collection of diagnostic, clinical and outcome data concerning the subject, which is kept without identification (coded) in a national research database of infants with liver disease. Samples of blood will be obtained for later research analysis, whenever possible, at the time of clinically indicated blood draws or when there is IV access for a clinical procedure. When liver biopsy specimens are obtained for diagnostic purposes, any liver biopsy specimen in excess of that needed for diagnostic use will be sent to the tissue repository. When a portoenterostomy or liver transplant occurs, sections of the liver and, biliary remnant removed in the course of surgery and in excess of that needed for diagnostic use, will be sent for the repository. These specimens will be used in investigations into the mechanisms and causes of the liver damage that occur in the participant's condition. . All data from this study will be kept in a secure research database at the Scientific Data Coordinating Center (SDCC) and transferred to the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) data repository after the study ends.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 1000
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in disease severity over time (disease progression) Measured at baseline, 1month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, then annually through year 15. disease progression defined by transplant date, date of death, worsening liver function, and complications related to worsening liver function
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (16)
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
University of California
🇺🇸San Francisco, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Children's Healthcare of Atlanta - Emory University
🇺🇸Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Riley Hospital for Children
🇺🇸Indianapolis, Indiana, United States
Johns Hopkins School of Medicine
🇺🇸Baltimore, Maryland, United States
Washington University School of Medicine
🇺🇸St Louis, Missouri, United States
Mount Sinai Medical Center
🇺🇸New York City, New York, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Baylor College of Medicine
🇺🇸Houston,, Texas, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada