Biliary Atresia Study in Infants and Children
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Biliary Atresia. Basic parameters: 6 months — 20 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Canada
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Biliary Atresia Study in Infants and Children (BASIC)
Overview
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.
Primary outcome measures
- 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.]
Secondary outcome measures (1)
- 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.]
Eligibility criteria
Inclusion criteria
- 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).
- 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).
- Participants with their native liver.
- 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
- Currently participating in the ChiLDReN study PROBE.
- Inability to confirm original diagnostic evaluation of biliary atresia.
- Inability or unwillingness of family or participant to participate in all scheduled visits.
- History of liver transplantation.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 15 centers
- Children's Hospital of Los Angeles — Los Angeles
- University of California at San Francisco — San Francisco
- Children's Hospital Colorado — Aurora
- Children's Healthcare of Atlanta - Emory University — Atlanta
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago
- Riley Children's Hospital — Indianapolis
- Johns Hopkins School of Medicine — Baltimore
- Washington University School of Medicine — St Louis
- … and 7 more centers
Canada · 1 center
- Hospital for Sick Children — Toronto
Publications
- Ng VL, Haber BH, Magee JC, Miethke A, Murray KF, Michail S, Karpen SJ, Kerkar N, Molleston JP, Romero R, Rosenthal P, Schwarz KB, Shneider BL, Turmelle YP, Alonso EM, Sherker AH, Sokol RJ; Childhood Liver Disease Research and Education Network (CHiLDREN). Medical status of 219 children with biliary atresia surviving long-term with their native livers: results from a North American multicenter cons PMID 25015575
Identifiers
NCT: NCT00345553 · BASIC Study - ChiLDReN Network · U01DK103149 · U01DK103140 · U01DK103135 · U01DK084575 · U01DK084538 · U01DK084536 · U01DK062503 · U01DK062500 · U01DK062497 · U01DK062481 · U01DK062470 · U01DK062466 · U01DK062456 · U01DK062453 · U01DK062452 · U01DK062445 · U01DK062436 · U24DK062456