NCT00061828

Brief Summary

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.

Trial Health

80
On Track

Trial Health Score

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

Enrollment
1,000

participants targeted

Target at P75+ for all trials

Timeline
38mo left

Started Apr 2004

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 Progress88%
Apr 2004May 2029

First Submitted

Initial submission to the registry

June 5, 2003

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 6, 2003

Completed
11 months until next milestone

Study Start

First participant enrolled

April 21, 2004

Completed
25.1 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

25.1 years

First QC Date

June 5, 2003

Last Update Submit

October 14, 2025

Conditions

Outcome Measures

Primary Outcomes (1)

  • Change in disease severity over time (disease progression)

    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.

Study Arms (1)

Biliary Atresia

Infants presenting with cholestasis who are diagnosed with biliary atresia.

Eligibility Criteria

AgeUp to 6 Months
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • 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.

You may not qualify if:

  • 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).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (16)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

University of California

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 Hospital for Children

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

Cincinnati 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

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

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

COMPLETED

Related Publications (9)

  • Teckman J, Rosenthal P, Ignacio RV, Spino C, Bass LM, Horslen S, Wang K, Magee JC, Karpen S, Asai A, Molleston JP, Squires RH, Kamath BM, Guthery SL, Loomes KM, Shneider BL, Sokol RJ; ChiLDReN (Childhood Liver Disease Research Network). Neonatal cholestasis in children with Alpha-1-AT deficiency is a risk for earlier severe liver disease with male predominance. Hepatol Commun. 2023 Dec 7;7(12):e0345. doi: 10.1097/HC9.0000000000000345. eCollection 2023 Dec 1.

  • Kemme S, Canniff JD, Feldman AG, Garth KM, Li S, Pan Z, Sokol RJ, Weinberg A, Mack CL. Cytomegalovirus in biliary atresia is associated with increased pretransplant death, but not decreased native liver survival. Hepatol Commun. 2023 Jul 17;7(8):e0175. doi: 10.1097/HC9.0000000000000175. eCollection 2023 Aug 1.

  • Hertel PM, Hawthorne K, Kim S, Finegold MJ, Shneider BL, Squires JE, Gupta NA, Bull LN, Murray KF, Kerkar N, Ng VL, Molleston JP, Bezerra JA, Loomes KM, Taylor SA, Schwarz KB, Turmelle YP, Rosenthal P, Magee JC, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Presentation and Outcomes of Infants With Idiopathic Cholestasis: A Multicenter Prospective Study. J Pediatr Gastroenterol Nutr. 2021 Oct 1;73(4):478-484. doi: 10.1097/MPG.0000000000003248.

  • Ng VL, Sorensen LG, Alonso EM, Fredericks EM, Ye W, Moore J, Karpen SJ, Shneider BL, Molleston JP, Bezerra JA, Murray KF, Loomes KM, Rosenthal P, Squires RH, Wang K, Arnon R, Schwarz KB, Turmelle YP, Haber BH, Sherker AH, Magee JC, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Neurodevelopmental Outcome of Young Children with Biliary Atresia and Native Liver: Results from the ChiLDReN Study. J Pediatr. 2018 May;196:139-147.e3. doi: 10.1016/j.jpeds.2017.12.048. Epub 2018 Mar 5.

  • Shneider BL, Magee JC, Karpen SJ, Rand EB, Narkewicz MR, Bass LM, Schwarz K, Whitington PF, Bezerra JA, Kerkar N, Haber B, Rosenthal P, Turmelle YP, Molleston JP, Murray KF, Ng VL, Wang KS, Romero R, Squires RH, Arnon R, Sherker AH, Moore J, Ye W, Sokol RJ; Childhood Liver Disease Research Network (ChiLDReN). Total Serum Bilirubin within 3 Months of Hepatoportoenterostomy Predicts Short-Term Outcomes in Biliary Atresia. J Pediatr. 2016 Mar;170:211-7.e1-2. doi: 10.1016/j.jpeds.2015.11.058. Epub 2015 Dec 24.

  • Ye W, Rosenthal P, Magee JC, Whitington PF; Childhood Liver Disease Research and Education Network. Factors Determining delta-Bilirubin Levels in Infants With Biliary Atresia. J Pediatr Gastroenterol Nutr. 2015 May;60(5):659-63. doi: 10.1097/MPG.0000000000000690.

  • Bessho K, Mourya R, Shivakumar P, Walters S, Magee JC, Rao M, Jegga AG, Bezerra JA. Gene expression signature for biliary atresia and a role for interleukin-8 in pathogenesis of experimental disease. Hepatology. 2014 Jul;60(1):211-23. doi: 10.1002/hep.27045. Epub 2014 May 27.

  • Shneider BL, Abel B, Haber B, Karpen SJ, Magee JC, Romero R, Schwarz K, Bass LM, Kerkar N, Miethke AG, Rosenthal P, Turmelle Y, Robuck PR, Sokol RJ; Childhood Liver Disease Research and Education Network. Portal hypertension in children and young adults with biliary atresia. J Pediatr Gastroenterol Nutr. 2012 Nov;55(5):567-73. doi: 10.1097/MPG.0b013e31826eb0cf.

  • Zahm AM, Hand NJ, Boateng LA, Friedman JR. Circulating microRNA is a biomarker of biliary atresia. J Pediatr Gastroenterol Nutr. 2012 Oct;55(4):366-9. doi: 10.1097/MPG.0b013e318264e648.

Related Links

Biospecimen

Retention: SAMPLES WITH DNA

In this iteration of the study protocol only samples of blood will be collected for research purposes.

MeSH Terms

Conditions

Biliary Atresia

Condition Hierarchy (Ancestors)

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

Study Officials

  • Saul Karpen, MD, PhD

    VCU School of Medicine

    STUDY CHAIR
  • Ed Doo, MD

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

    STUDY DIRECTOR
  • John Magee, MD

    University of Michigan Medical Center, Ann Arbor

    PRINCIPAL INVESTIGATOR
  • Lisa Henn, PhD

    Arbor Research Collaborative for Health

    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 5, 2003

First Posted

June 6, 2003

Study Start

April 21, 2004

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