A Prospective Database of Infants With Cholestasis (PROBE)

June 3, 2026 updated by: Arbor Research Collaborative for Health

Childhood Liver Disease Research Network (ChiLDReN): A Prospective Database of Infants With Cholestasis

Biliary atresia, idiopathic neonatal hepatitis, and specific genetic cholestatic conditions are the most common causes of jaundice and hyperbilirubinemia that continue beyond the newborn period. The long term goal of the Childhood Liver Disease Research Network (ChiLDReN) is to establish a database of clinical information and plasma, serum, and tissue samples from cholestatic children to facilitate research and to perform clinical, epidemiological and therapeutic trials in these important pediatric liver diseases.

Study Overview

Status

Recruiting

Conditions

Detailed Description

This is a multi-center project to establish a prospective database of clinical information and a repository of blood, from children with diagnosis of neonatal liver disease, such as biliary atresia (BA), in order to perform research in this important liver problem. Children (diagnosed with BA or suspicious for BA) will be screened and enrolled at presentation at the participating pediatric liver sites. Participants diagnosed with BA will be followed intensively for the first year, at 18 months of age, and then annually up to 10 years of age, and then biannually, or liver transplantation. Other participants (Non-BA) diagnosed with cholestasis will be exited from the study at the time of diagnosis determination.

Detailed clinical data, laboratory investigations, liver and biliary specimens, and long-term follow-up of outcomes are part of the normal standard of care with respect to the diagnosis and treatment of the subjects with liver problems. This research involves the collection of diagnostic, clinical and outcome data concerning the subject, which is kept without identification (coded) in a national research database of infants with liver disease. Samples of blood will be obtained for later research analysis, whenever possible, at the time of clinically indicated blood draws or when there is IV access for a clinical procedure. All data from this study will be kept in a secure research database at the Scientific Data Coordinating Center (SDCC) and transferred to the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) data repository after the study ends.

Study Type

Observational

Enrollment (Estimated)

1000

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
        • Completed
        • The Hospital for Sick Children
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital Los Angeles
        • Contact:
        • Principal Investigator:
          • Rohit Kohli, MD
        • Sub-Investigator:
          • Christopher Gayer, MD
      • San Francisco, California, United States, 94143
        • Completed
        • University of California
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Children's Hospital Colorado
        • Contact:
        • Principal Investigator:
          • Ronald Sokol, MD
        • Sub-Investigator:
          • Michael Narkewicz, MD
        • Sub-Investigator:
          • Amy Feldman, MD
        • Sub-Investigator:
          • Dania Brigham, MD
        • Sub-Investigator:
          • Shikha Sundaram, MD
        • Contact:
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Children's Healthcare of Atlanta - Emory University
        • Principal Investigator:
          • Nitika Gupta, MD
        • Sub-Investigator:
          • Miriam Vos, MD MSPH
        • Contact:
        • Contact:
        • Sub-Investigator:
          • Rene Romero, MD
        • Sub-Investigator:
          • Saul Karpen, MD, PhD
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Recruiting
        • Ann & Robert H. Lurie Children's Hospital of Chicago
        • Contact:
        • Principal Investigator:
          • Estella Alonso, MD
        • Sub-Investigator:
          • Lee Bass, MD
        • Contact:
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Completed
        • Riley Hospital for Children
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Completed
        • Johns Hopkins School of Medicine
    • Missouri
      • St 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
        • Cincinnati 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
        • Completed
        • UPMC Children's Hospital of Pittsburgh
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • 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
        • Contact:
        • Principal Investigator:
          • Stephen Guthery, MD
        • Sub-Investigator:
          • Kyle Jensen, MD
        • Sub-Investigator:
          • Linda Book, MD
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98105
        • Recruiting
        • Seattle Children's Hospital
        • Principal Investigator:
          • Pamela Valentino, MD
        • Contact:
        • Sub-Investigator:
          • Evelyn Hsu, MD
        • Sub-Investigator:
          • Niviann Blondet, MD
        • Contact:

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

No older than 6 months (Child)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

This study population will be selected from the patient base of participating specialty care clinics.

Description

INCLUSION CRITERIA

  • Infant's age less than or equal to 180 days at initial presentation at the ChiLDReN clinical site.
  • Diagnosis of cholestasis defined by serum direct or conjugated bilirubin greater than or equal to 2 mg/dl and suspected biliary atresia.
  • The subject's parent(s)/guardian(s) willing to provide informed written consent.

EXCLUSION CRITERIA

  • Acute liver failure.
  • Previous hepatobiliary surgery with dissection or excision of biliary tissue.
  • Diagnoses of bacterial or fungal sepsis (except where associated with metabolic liver disease)
  • Diagnoses of hypoxia, shock or ischemic hepatopathy within the past two weeks (If the cholestasis persists beyond two weeks of the initiating event, the infant can be enrolled).
  • Diagnosis of any malignancy.
  • Presence of any primary hemolytic disease (except when diagnosed with biliary atresia or another cholestatic disease being studied by ChiLDREN).
  • Diagnosis of any drug or Total parenteral nutrition (TPN)-associated cholestasis (except when diagnosed with biliary atresia or another cholestatic disease being studied by ChiLDREN).
  • Diagnosis with Extracorporeal membrane oxygenation (ECMO)-associated cholestasis.
  • Birth weight less than 1500g (except when diagnosed with biliary atresia).

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
Biliary Atresia
Infants presenting with cholestasis who are diagnosed with biliary atresia.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in disease severity over time (disease progression)
Time Frame: Measured at baseline, 1 month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, annually through year 10 and then biannually through year 20.
disease progression defined by transplant date, date of death, worsening liver function, and complications related to worsening liver function
Measured at baseline, 1 month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, annually through year 10 and then biannually through year 20.

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

  • Principal Investigator: John Magee, MD, University of Michigan Medical Center, Ann Arbor
  • Study Director: Ed Doo, MD, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • Principal Investigator: Lisa Henn, PhD, Arbor Research Collaborative for Health
  • Study Director: Katrina Loh, MD, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
  • Study Chair: Saul Karpen, MD, PhD, VCU School of Medicine

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)

April 21, 2004

Primary Completion (Estimated)

May 31, 2029

Study Completion (Estimated)

May 31, 2029

Study Registration Dates

First Submitted

June 5, 2003

First Submitted That Met QC Criteria

June 5, 2003

First Posted (Estimated)

June 6, 2003

Study Record Updates

Last Update Posted (Actual)

June 4, 2026

Last Update Submitted That Met QC Criteria

June 3, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PROBE 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)
  • U24DK062456 (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

product manufactured in and exported from the U.S.

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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