Biliary Atresia Study in Infants and Children (BASIC)

September 12, 2023 updated by: Arbor Research Collaborative for Health

Biliary Atresia Study in Infants and Children (BASIC)

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.

Study Overview

Status

Recruiting

Conditions

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 Type

Observational

Enrollment (Estimated)

1265

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Recruiting
        • Hospital for Sick Children
        • Sub-Investigator:
          • Vicki Ng, MD
        • Contact:
        • Principal Investigator:
          • Binita Kamath, MD
        • Contact:
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital of Los Angeles
        • Contact:
        • Sub-Investigator:
          • Danny Thomas, MD
        • Sub-Investigator:
          • Nisreen Soufi, MD
        • Sub-Investigator:
          • Sonia Michail, MD
        • Principal Investigator:
          • Rohit Kohli, MD
      • San Francisco, California, United States, 94143
        • Active, not recruiting
        • University of California at San Francisco
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Children's Hospital Colorado
        • Contact:
        • Contact:
        • Principal Investigator:
          • Ronald J Sokol, MD
        • Sub-Investigator:
          • Michael Narkewicz, MD
        • Sub-Investigator:
          • Amy Feldman, MD
        • Sub-Investigator:
          • Dania Brigham, MD
        • Sub-Investigator:
          • Shikha Sundaram, MD
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Children's Healthcare of Atlanta - Emory University
        • Principal Investigator:
          • Saul Karpen, MD PhD
        • Sub-Investigator:
          • Miriam Vos, MD
        • Contact:
        • Contact:
        • Sub-Investigator:
          • Nitika Gupta, MD
        • Sub-Investigator:
          • Rene Romero, MD
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Recruiting
        • Ann & Robert H. Lurie Children's Hospital of Chicago
        • Contact:
        • Contact:
        • Principal Investigator:
          • Estella Alonso, MD
        • Sub-Investigator:
          • Lee Bass, MD
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Riley Children's Hospital
        • Principal Investigator:
          • Jean Molleston, MD
        • Sub-Investigator:
          • Molly Bozic, MD
        • Contact:
        • Contact:
          • Ann Klipsch, RN
          • Phone Number: 317-274-9605
          • Email: aeye@iu.edu
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Completed
        • Johns Hopkins School of Medicine
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Completed
        • Washington University School of Medicine
    • New York
      • New York, New York, United States, 10029
        • Completed
        • Mount Sinai Medical Center
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Children's Hospital Medical Center
        • Contact:
        • Contact:
        • Sub-Investigator:
          • Joseph Palermo, MD
        • Principal Investigator:
          • Alexander Miethke, MD
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Principal Investigator:
          • Kathleen Loomes, MD
        • Sub-Investigator:
          • David Piccoli, MD
        • Sub-Investigator:
          • Elizabeth Rand, MD
        • Contact:
        • Contact:
      • Pittsburgh, Pennsylvania, United States, 15224
        • Recruiting
        • UPMC Children's Hospital of Pittsburgh
        • Sub-Investigator:
          • James Squires, MD
        • Principal Investigator:
          • Simon Horslen, MD
        • Contact:
        • Contact:
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Texas Children's Hospital (Baylor College of Medicine)
        • Principal Investigator:
          • Paula Hertel, MD
        • Sub-Investigator:
          • Benjamin Shneider, MD
        • Contact:
        • Contact:
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Recruiting
        • University of Utah
        • Principal Investigator:
          • Kyle Jensen, MD
        • Sub-Investigator:
          • Stephen Guthery, MD
        • Contact:
        • Sub-Investigator:
          • Linda Book, MD
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98105
        • Recruiting
        • Seattle Children's Hospital
        • Principal Investigator:
          • Pamela Vaentino, MD
        • Contact:
        • Sub-Investigator:
          • Evelyn Hsu, MD
        • Sub-Investigator:
          • Niviann Blondet, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 20 years (Child, Adult)

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Specimens for this aim are collected once during study, usually at baseline.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
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.
Observational information collected at entrance into study as well as at each yearly follow-up visit.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Arbor Research Collaborative for Health

Collaborators

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)
  • Study Director: Averell Sherker, MD, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • Principal Investigator: John C Magee, MD, University of Michigan
  • Principal Investigator: Lisa Henn, PhD, Arbor Research Collaborative for Health - Data Coordinating Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 16, 2006

Primary Completion (Estimated)

May 1, 2024

Study Completion (Estimated)

May 1, 2024

Study Registration Dates

First Submitted

June 27, 2006

First Submitted That Met QC Criteria

June 27, 2006

First Posted (Estimated)

June 28, 2006

Study Record Updates

Last Update Posted (Actual)

September 13, 2023

Last Update Submitted That Met QC Criteria

September 12, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • BASIC Study - ChiLDReN Network
  • U01DK062436 (U.S. NIH Grant/Contract)
  • U01DK062456 (U.S. NIH Grant/Contract)
  • 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)
  • U01DK062453 (U.S. NIH Grant/Contract)
  • U01DK062452 (U.S. NIH Grant/Contract)
  • U01DK062445 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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