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Summary

for people ages 6 months and up (full criteria)
at San Francisco, California and other locations
study started
estimated completion:

Description

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.

Official Title

Biliary Atresia Study in Infants and Children (BASIC)

Details

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 Biliary Atresia Research Clinical Research Consortium (BARC), an NIDDK-funded network.

Keywords

Biliary Atresia Cholestasis

Eligibility

You can join if…

Open to people ages 6 months and up

  1. Subjects 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. Subjects need to be greater than or equal to (with no upper age limit).
  3. Subject either have their native liver or have a confirmed liver transplantation.
  4. Parent, guardian or subject (if 18 years of age or older) is willing to provide informed consent and, when appropriate, the subject is willing to assent.

You CAN'T join if...

  1. Enrollment in the PROBE study
  2. Inability to confirm original diagnostic evaluation of biliary atresia
  3. Inability or unwillingness of family or subject to participate in all scheduled visits.

Locations

  • Children's Hospital of Los Angeles accepting new patients
    Los Angeles, California, 90027, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
ID
NCT00345553
Study Type
Observational
Last Updated
September 1, 2016