Biliary Atresia Study in Infants and Children

Recruiting

• Conditions: Biliary Atresia

• Registration Number: NCT00345553
• Lead Sponsor: Arbor Research Collaborative for Health

Brief Summary

Little is known about the factors that cause biliary atresia nor the factors that influence disease progression. The purpose of this study is to collect the pertinent clinical information, genetic material and body fluid samples to enable investigators to address the following aims: To identify the gene or genes implicated in the etiology of BA; To characterize the natural history of the older, non-transplanted child with BA.

Detailed Description

Little is known about the factors that cause biliary atresia nor the factors that influence disease progression. A variety of genetic, autoimmune and environmental influences have been hypothesized to be important. Most studies to date have focused on the neonate and young child with BA, yet the older surviving child with BA can provide important information about genetics, as well as, natural history.

The purpose of this study is to collect the pertinent clinical information, genetic material and body fluid samples to enable investigators to address the following hypotheses:

Hypothesis 1: A genetic defect is a likely causative factor for BA among children with BA and multiple congenital anomalies.

Hypothesis 2a: Sentinel events such as variceal bleeding, ascites and growth failure are earlier predictors of death or need for liver transplantation than the pediatric end-stage liver disease score (PELD).

Hypothesis 2b: Health related quality of life will be impaired compared to healthy age matched children and relate to severity of illness.

Hypothesis 2c: Growth failure as measured by anthropometrics and nutritional supplementation will be predictive of onset of sentinel events (ascites, variceal bleed, death, and transplant) in the following 24 months.

This study will be performed by the Childhood Liver Disease Research Network (ChiLDReN), a National Institute of Diabetes \& Digestive and Kidney Diseases (NIDDK) funded network.

Study Timeline

• Study Start: 2006-05-16
• Study First Submitted: 2006-06-27
• Study First Submitted QC: 2006-06-27
• Study First Posted: 2006-06-28
• Status Verified: 2025-09
• Last Update Submitted: 2025-09-19
• Last Update Posted: 2025-09-24
• Primary Completion: 2029-05-31
• Study Completion: 2029-05-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 1265

Inclusion Criteria

1. Participants need to have a confirmed diagnosis of BA determined by chart review including review of pertinent diagnostic biopsy reports, radiologic reports and surgical reports (if surgery was performed).
2. Participants need to be >6 months of age up to and equal to the age of 20 (participants enrolled at 20 years of age will have one visit).
3. Participants with their native liver.
4. Parent, guardian or participant (if 18 years of age or older) is willing to provide informed consent and, when appropriate, the participant is willing to assent.

Exclusion Criteria

1. Currently participating in the ChiLDReN study PROBE.
2. Inability to confirm original diagnostic evaluation of biliary atresia.
3. Inability or unwillingness of family or participant to participate in all scheduled visits.
4. History of liver transplantation.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To identify the gene or genes implicated in the etiology of BA	Specimens for this aim are collected once during study, usually at baseline.	The genetics of BA may be investigated on two levels. The first is to identify a group of patients whose etiology is a result of a genetic defect and the second is to examine the influence of genetics on disease acquisition.

Secondary Outcome Measures

Name	Time	Method
Define the natural history of the older, non-transplanted child with biliary atresia	Observational information collected at entrance into study as well as at each yearly follow-up visit.	Understanding the natural history of a disease is a prerequisite to interpreting disease severity, identifying patterns of illness, identifying early predictors of outcome and understanding the advantages or trade-offs of therapeutic interventions.

Trial Locations

Locations (16)

Children's Hospital of Los Angeles; 🇺🇸 Los Angeles, California, United States

University of California at San Francisco; 🇺🇸 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 Children's Hospital; 🇺🇸 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, New York, United States

Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

Scroll for more (6 remaining)