Biliary Atresia Study in Infants and Children (BASIC)

• ClinicalTrials.gov Identifier: NCT00345553
• Recruitment Status: Suspended
• First Posted: June 28, 2006
• Last Update Posted: April 3, 2020
• Sponsor: Arbor Research Collaborative for Health
• Collaborator: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Information provided by (Responsible Party): Arbor Research Collaborative for Health

Study Description

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.

Condition or disease
Biliary Atresia

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 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 Design

• Study Type: Observational
• Estimated Enrollment: 1265 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• 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

Groups and Cohorts

Group/Cohort
1; Biliary atresia subjects who have their native liver
2; Biliary atresia subjects who have had a liver transplant

Outcome Measures

Primary Outcome Measures :

1. 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.

Secondary Outcome Measures :

1. 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. ]
2. 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

Samples of blood will be collected for research purposes.

Eligibility Criteria

• Ages Eligible for Study: 6 Months to 20 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

Recruitment will be open to any eligible subject. The method of contacting the study subjects will conform to local IRB guidelines, but in general: the parents or guardians of all eligible subjects at each ChiLDReN center, or the subjects themselves if 18 years of age or older, will be approached to participate. New patients who are not participating in ChiLDReN study PROBE may be approached for study participation, but will not be eligible for enrollment until 6 months of age.

Criteria

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.

Contacts and Locations

Locations

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

Canada, Ontario

Hospital for Sick Children

Toronto, Ontario, Canada, M5G 1X8

Sponsors and Collaborators

Arbor Research Collaborative for Health

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Investigators

• 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)

More Information

Additional Information:

Childhood Liver Disease Research Network (ChiLDReN) website 

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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 consortium. J Pediatr. 2014 Sep;165(3):539-546.e2. doi: 10.1016/j.jpeds.2014.05.038. Epub 2014 Jul 9. Erratum in: J Pediatr. 2015 Jan;166(1):211.

• Responsible Party: Arbor Research Collaborative for Health
• ClinicalTrials.gov Identifier: NCT00345553
• Other Study ID Numbers: BASIC Study - ChiLDReN Network; U01DK103149 ( U.S. NIH Grant/Contract ); U01DK103140 ( U.S. NIH Grant/Contract ); U01DK103135 ( U.S. NIH Grant/Contract ); U01DK084575 ( U.S. NIH Grant/Contract ); U01DK084538 ( U.S. NIH Grant/Contract ); U01DK084536 ( U.S. NIH Grant/Contract ); U01DK062503 ( U.S. NIH Grant/Contract ); U01DK062500 ( U.S. NIH Grant/Contract ); U01DK062497 ( U.S. NIH Grant/Contract ); U01DK062481 ( U.S. NIH Grant/Contract ); U01DK062470 ( U.S. NIH Grant/Contract ); U01DK062466 ( U.S. NIH Grant/Contract ); U01DK062456 ( U.S. NIH Grant/Contract ); U01DK062453 ( U.S. NIH Grant/Contract ); U01DK062452 ( U.S. NIH Grant/Contract ); U01DK062445 ( U.S. NIH Grant/Contract ); U01DK062436 ( U.S. NIH Grant/Contract )
• First Posted: June 28, 2006
• Last Update Posted: April 3, 2020
• Last Verified: February 2020

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: The data will be transferred to NIDDK at the end of the study.

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Arbor Research Collaborative for Health:

Biliary Atresia; Cholestasis

Additional relevant MeSH terms:

Biliary Atresia; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Digestive System Abnormalities; Congenital Abnormalities