A Prospective Database of Infants With Cholestasis

Biliary Atresia Clinical Trial

— PROBE  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00061828
• Other study ID #: PROBE Study - ChiLDReN Network
• Secondary ID: U01DK103149U01DK
• Status: Recruiting
• Start date: April 21, 2004
• Est. completion date: May 2029

Study information

• Verified date: June 2024
• Source: Arbor Research Collaborative for Health
• Contact: Terese A Howell
• Phone: 734 476-5340
• Email: terri.howell[@]arborresearch.org
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Description:

This is a multi-center project to establish a prospective database of clinical information and a repository of blood, and tissue samples from children with diagnoses of neonatal liver diseases, such as biliary atresia (BA), idiopathic neonatal hepatitis (INH), and specific neonatal presentations of genetic cholestatic disorders in order to perform research in these important liver problems. Children 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 20 years of age, or liver transplantation. Other participants diagnosed with cholestasis will be followed on the same schedule; if there is complete (clinical and biochemical) resolution of their underlying liver disease off all therapy, there will be one follow up visit within one year (preferably scheduled at the time of the next planned follow up visit or at 12 months of age, whichever is later) for data collection and to obtain blood samples. The development of a serum and tissue bank of specimens from children with various neonatal cholestatic disorders will be an invaluable tool for current and future investigations into the etiology and pathogenesis of hepatobiliary injury in the infant.

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. When liver biopsy specimens are obtained for diagnostic purposes, any liver biopsy specimen in excess of that needed for diagnostic use will be sent to the tissue repository. When a portoenterostomy or liver transplant occurs, sections of the liver and, biliary remnant removed in the course of surgery and in excess of that needed for diagnostic use, will be sent for the repository. These specimens will be used in investigations into the mechanisms and causes of the liver damage that occur in the participant's condition. . 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1000
• Est. completion date: May 2029
• Est. primary completion date: May 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 6 Months

Eligibility

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 20% of total and greater than or equal to 2 mg/dl.

• 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).

Related Conditions & MeSH terms

• Biliary Atresia
• Cholestasis
• Neonatal Cholestasis

Locations

Country	Name	City	State
Canada	The Hospital for Sick Children	Toronto	Ontario
United States	Children's Healthcare of Atlanta - Emory University	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Johns Hopkins School of Medicine	Baltimore	Maryland
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Baylor College of Medicine	Houston	Texas
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	Mount Sinai Medical Center	New York	New York
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah
United States	University of California	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington

Sponsors (2)

Lead Sponsor	Collaborator
Arbor Research Collaborative for Health	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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, 1month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, then annually through year 15.

External Links

•   Childhood Liver Disease Research Network (ChiLDReN) website