NCT00345553

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 characterize the natural history of the older, non-transplanted child with BA.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,265

participants targeted

Target at P75+ for all trials

Timeline
38mo left

Started May 2006

Longer than P75 for all trials

Geographic Reach
2 countries

16 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress87%
May 2006May 2029

Study Start

First participant enrolled

May 16, 2006

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

June 27, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 28, 2006

Completed
22.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 31, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 31, 2029

Last Updated

October 15, 2025

Status Verified

September 1, 2025

Enrollment Period

23.1 years

First QC Date

June 27, 2006

Last Update Submit

October 14, 2025

Conditions

Keywords

Biliary AtresiaCholestasis

Outcome Measures

Primary Outcomes (1)

  • To identify the gene or genes implicated in the etiology of BA

    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.

    Specimens for this aim are collected once during study, usually at baseline.

Secondary Outcomes (1)

  • Define the natural history of the older, non-transplanted child with biliary atresia

    Observational information collected at entrance into study as well as at each yearly follow-up visit.

Study Arms (1)

1

Biliary atresia subjects who have their native liver

Eligibility Criteria

Age6 Months - 20 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-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.

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (16)

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

University of California at San Francisco

San Francisco, California, 94143, United States

COMPLETED

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

RECRUITING

Children's Healthcare of Atlanta - Emory University

Atlanta, Georgia, 30322, United States

RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60614, United States

RECRUITING

Riley Children's Hospital

Indianapolis, Indiana, 46202, United States

COMPLETED

Johns Hopkins School of Medicine

Baltimore, Maryland, 21287, United States

COMPLETED

Washington University School of Medicine

St Louis, Missouri, 63110, United States

COMPLETED

Mount Sinai Medical Center

New York, New York, 10029, United States

COMPLETED

Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Texas Children's Hospital (Baylor College of Medicine)

Houston, Texas, 77030, United States

RECRUITING

University of Utah

Salt Lake City, Utah, 84113, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

COMPLETED

Related Publications (1)

  • 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.

Related Links

Biospecimen

Retention: SAMPLES WITH DNA

Samples of blood will be collected for research purposes.

MeSH Terms

Conditions

Biliary AtresiaCholestasis

Condition Hierarchy (Ancestors)

Bile Duct DiseasesBiliary Tract DiseasesDigestive System DiseasesDigestive System AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Sanjiv Harpavat, MD

    Texas Children's/Baylor College of Medicine

    STUDY CHAIR
  • Ed Doo, MD

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

    STUDY DIRECTOR
  • John C Magee, MD

    University of Michigan

    PRINCIPAL INVESTIGATOR
  • Lisa Henn, PhD

    Arbor Research Collaborative for Health - Data Coordinating Center

    PRINCIPAL INVESTIGATOR
  • Katrina Loh, MD

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

    STUDY DIRECTOR

Central Study Contacts

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 27, 2006

First Posted

June 28, 2006

Study Start

May 16, 2006

Primary Completion (Estimated)

May 31, 2029

Study Completion (Estimated)

May 31, 2029

Last Updated

October 15, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will share

The data will be transferred to NIDDK at the end of the study.

Locations