Biliary Atresia Study in Infants and Children

NCT ID: NCT00345553

Last Updated: 2025-10-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

1265 participants

Study Classification

OBSERVATIONAL

Study Start Date

2006-05-16

Study Completion Date

2029-05-31

Brief Summary

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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

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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.

Conditions

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Biliary Atresia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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1

Biliary atresia subjects who have their native liver

No interventions assigned to this group

Eligibility Criteria

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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 with their native liver.
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.
4. History of liver transplantation.
Minimum Eligible Age

6 Months

Maximum Eligible Age

20 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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

NIH

Sponsor Role collaborator

Arbor Research Collaborative for Health

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sanjiv Harpavat, MD

Role: STUDY_CHAIR

Texas Children's/Baylor College of Medicine

Ed Doo, MD

Role: STUDY_DIRECTOR

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

John C Magee, MD

Role: PRINCIPAL_INVESTIGATOR

University of Michigan

Lisa Henn, PhD

Role: PRINCIPAL_INVESTIGATOR

Arbor Research Collaborative for Health - Data Coordinating Center

Katrina Loh, MD

Role: STUDY_DIRECTOR

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

Locations

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Children's Hospital of Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

University of California at San Francisco

San Francisco, California, United States

Site Status COMPLETED

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status RECRUITING

Children's Healthcare of Atlanta - Emory University

Atlanta, Georgia, United States

Site Status RECRUITING

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

Chicago, Illinois, United States

Site Status RECRUITING

Riley Children's Hospital

Indianapolis, Indiana, United States

Site Status COMPLETED

Johns Hopkins School of Medicine

Baltimore, Maryland, United States

Site Status COMPLETED

Washington University School of Medicine

St Louis, Missouri, United States

Site Status COMPLETED

Mount Sinai Medical Center

New York, New York, United States

Site Status COMPLETED

Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

Texas Children's Hospital (Baylor College of Medicine)

Houston, Texas, United States

Site Status RECRUITING

University of Utah

Salt Lake City, Utah, United States

Site Status RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

Site Status RECRUITING

Hospital for Sick Children

Toronto, Ontario, Canada

Site Status COMPLETED

Countries

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United States Canada

Central Contacts

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Terese A Howell, BS

Role: CONTACT

734-476-5340

Melissa Sexton, BBA

Role: CONTACT

734-693-3811

Facility Contacts

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Sirikorn Boonsawat

Role: primary

323-361-2181

Kelly Gilmartin

Role: primary

720-777-2961

Michelle Landis

Role: backup

720-777-8884

Katelynn Harris

Role: primary

404-785-0421

Jordyn Turner

Role: backup

404-785-3690

Jacqueline Alexander

Role: primary

312-227-3523

Angela Anthony

Role: backup

312-227-4559

Jennifer Hawkins, MS

Role: primary

513-636-7818

Erin Chapman, BAS

Role: backup

513-803-7482

Caitlin Griffiths, MS

Role: primary

267-577-9888

Jordan Moreno

Role: backup

267-425-1614

Audrey Fitzgerald

Role: primary

412-692-3378

Susan Richey, RN

Role: backup

412-692-6337

Laurel Cavallo

Role: primary

832-822-1053

Cynthia Tsai

Role: backup

832-822-3634

Ann Rutherford

Role: primary

801-585-9495

Natalie Fillerup

Role: backup

801-587-5670

Melissa Young

Role: primary

206-987-1037

Teresa Chen

Role: backup

206-987-6649

References

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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.

Reference Type DERIVED
PMID: 25015575 (View on PubMed)

Related Links

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http://www.childrennetwork.org

Childhood Liver Disease Research Network (ChiLDReN) website

Other Identifiers

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U01DK103149

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK103140

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK103135

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK084575

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK084538

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK084536

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062503

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062500

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062497

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062481

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062470

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062466

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062456

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062453

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062452

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062445

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U01DK062436

Identifier Type: NIH

Identifier Source: secondary_id

View Link

U24DK062456

Identifier Type: NIH

Identifier Source: secondary_id

View Link

BASIC Study - ChiLDReN Network

Identifier Type: -

Identifier Source: org_study_id

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