Clinical Trial: Biliary Atresia Study in Infants and Children
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
Official Title: Biliary Atresia Study in Infants and Children (BASIC)
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 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.
Detailed Summary:
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 Researc
Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Current Primary Outcome: To identify the gene or genes implicated in the etiology of BA [ Time Frame: Specimens for this aim are collected once during study, usually at baseline. ]
Original Primary Outcome:
Current Secondary Outcome:
- To identify polymorphisms that may be important in disease progression such as HLA polymorphisms [ Time Frame: Specimens for this aim are collected once during study, usually at baseline. ]
- Define the natural history of the older, non-transplanted child with biliary atresia [ Time Frame: Observational information collected at entrance into study as well as at each yearly follow-up visit. ]
Original Secondary Outcome:
Information By: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Dates:
Date Received: June 27, 2006
Date Started: May 2006
Date Completion: May 2019
Last Updated: September 20, 2016
Last Verified: September 2016
