Biliary Atresia Study in Infants and Children (BASIC)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00345553|
Recruitment Status : Suspended (Study visits suspended due to COVID-19 pandemic.)
First Posted : June 28, 2006
Last Update Posted : April 3, 2020
|Condition or disease|
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.
|Study Type :||Observational|
|Estimated Enrollment :||1265 participants|
|Official Title:||Biliary Atresia Study in Infants and Children (BASIC)|
|Study Start Date :||May 2006|
|Estimated Primary Completion Date :||May 2024|
|Estimated Study Completion Date :||May 2024|
Biliary atresia subjects who have their native liver
Biliary atresia subjects who have had a liver transplant
- 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. ]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.
- To identify polymorphisms that may be important in disease progression such as Human leukocyte antigen (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. ]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.
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00345553
|United States, California|
|Children's Hospital of Los Angeles|
|Los Angeles, California, United States, 90027|
|University of California at San Francisco|
|San Francisco, California, United States, 94143|
|United States, Colorado|
|Children's Hospital Colorado|
|Aurora, Colorado, United States, 80045|
|United States, Georgia|
|Children's Healthcare of Atlanta - Emory University|
|Atlanta, Georgia, United States, 30322|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60614|
|United States, Indiana|
|Riley Children's Hospital|
|Indianapolis, Indiana, United States, 46202|
|United States, Maryland|
|Johns Hopkins School of Medicine|
|Baltimore, Maryland, United States, 21287|
|United States, Missouri|
|Washington University School of Medicine|
|Saint Louis, Missouri, United States, 63110|
|United States, New York|
|Mount Sinai Medical Center|
|New York, New York, United States, 10029|
|United States, Ohio|
|Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|UPMC Children's Hospital of Pittsburgh|
|Pittsburgh, Pennsylvania, United States, 15224|
|United States, Texas|
|Texas Children's Hospital (Baylor College of Medicine)|
|Houston, Texas, United States, 77030|
|United States, Utah|
|University of Utah|
|Salt Lake City, Utah, United States, 84113|
|United States, Washington|
|Seattle Children's Hospital|
|Seattle, Washington, United States, 98105|
|Hospital for Sick Children|
|Toronto, Ontario, Canada, M5G 1X8|
|Study Chair:||Vicky Ng, MD||The Hospital for Sick Children, Toronto, ON, Canada|
|Study Director:||Ed Doo, MD||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|
|Principal Investigator:||John C Magee, MD||University of Michigan|
|Principal Investigator:||Robert M Merion, MD||Arbor Research Collaborative for Health - Data Coordinating Center|
|Study Director:||Averell Sherker, MD||National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|