Biliary Atresia Study in Infants and Children

Biliary Atresia Clinical Trial

— BASIC  

Official title:

Biliary Atresia Study in Infants and Children (BASIC)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00345553
• Other study ID #: BASIC Study - ChiLDReN Network
• Secondary ID: U01DK103149U01DK
• Status: Recruiting
• Start date: May 16, 2006
• Est. completion date: May 2029

Study information

• Verified date: June 2024
• Source: Arbor Research Collaborative for Health
• Contact: Terese A Howell, BS
• Phone: 734-476-5340
• Email: terri.howell[@]arborresearch.org
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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 identify polymorphisms that may be important in disease progression such as HLA polymorphisms; To characterize the natural history of the older, non-transplanted child with BA.

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 2: Autoimmune factors are likely to contribute to disease progression or acquisition and can be identified by correlating HLA among children with BA to healthy controls and by comparison of those who develop early complications including, variceal bleed, ascites, and growth failure compared to those who do not.

Hypothesis 3a: 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 3b: Health related quality of life will be impaired compared to healthy age matched children and relate to severity of illness.

Hypothesis 3c: 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1265
• Est. completion date: May 2029
• Est. primary completion date: May 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 20 Years

Eligibility

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 either have their native liver or have a confirmed liver transplantation.

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.

Related Conditions & MeSH terms

• Biliary Atresia

Locations

Country	Name	City	State
Canada	Hospital for Sick Children	Toronto	Ontario
United States	Children's Healthcare of Atlanta - Emory University	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Johns Hopkins School of Medicine	Baltimore	Maryland
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Children's Hospital Medical Center	Cincinnati	Ohio
United States	Texas Children's Hospital (Baylor College of Medicine)	Houston	Texas
United States	Riley Children's Hospital	Indianapolis	Indiana
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	Mount Sinai Medical Center	New York	New York
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah
United States	University of California at San Francisco	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (2)

Lead Sponsor	Collaborator
Arbor Research Collaborative for Health	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To identify the gene or genes implicated in the etiology of BA	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.	Specimens for this aim are collected once during study, usually at baseline.
Secondary	To identify polymorphisms that may be important in disease progression such as Human leukocyte antigen (HLA) polymorphisms		Specimens for this aim are collected once during study, usually at baseline.
Secondary	Define the natural history of the older, non-transplanted child with biliary atresia	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.	Observational information collected at entrance into study as well as at each yearly follow-up visit.

External Links

•   Childhood Liver Disease Research Network (ChiLDReN) website