Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Prospective Database of Infants With Cholestasis (PROBE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00061828
Recruitment Status : Suspended (Study visits suspended due to COVID-19 pandemic.)
First Posted : June 6, 2003
Last Update Posted : April 2, 2020
Sponsor:
Collaborator:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by (Responsible Party):
Arbor Research Collaborative for Health

Tracking Information
First Submitted Date June 5, 2003
First Posted Date June 6, 2003
Last Update Posted Date April 2, 2020
Study Start Date May 2004
Estimated Primary Completion Date May 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 28, 2016)
Change in disease severity over time (disease progression) [ Time Frame: Measured at baseline, 1month, 2 months, 3, 6 months post-baseline, 12 and 18 months of age, then annually through year 15. ]
disease progression defined by transplant date, date of death, worsening liver function, and complications related to worsening liver function
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Prospective Database of Infants With Cholestasis
Official Title Childhood Liver Disease Research Network (ChiLDReN): A Prospective Database of Infants With Cholestasis
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.
Detailed 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.

Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Samples of blood, and liver tissue samples will be collected for research purposes.
Sampling Method Non-Probability Sample
Study Population This study population will be selected from the patient base of participating specialty care clinics.
Condition
  • Biliary Atresia
  • Neonatal Cholestasis
Intervention Not Provided
Study Groups/Cohorts
  • Biliary Atresia
    Infants presenting with cholestasis who are diagnosed with biliary atresia.
  • Non-Biliary Atresia
    Infants presenting with cholestasis without a diagnosis of biliary atresia.
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Suspended
Estimated Enrollment
 (submitted: April 23, 2012)
1000
Original Enrollment
 (submitted: June 23, 2005)
400
Estimated Study Completion Date May 2024
Estimated Primary Completion Date May 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria

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).
Sex/Gender
Sexes Eligible for Study: All
Ages up to 6 Months   (Child)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00061828
Other Study ID Numbers PROBE Study - ChiLDReN Network
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 )
U01DK062456 ( U.S. NIH Grant/Contract )
U01DK062453 ( U.S. NIH Grant/Contract )
U01DK062452 ( U.S. NIH Grant/Contract )
U01DK062445 ( U.S. NIH Grant/Contract )
U01DK062436 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: Yes
Plan Description: The data will be transferred to NIDDK at the end of the study.
Responsible Party Arbor Research Collaborative for Health
Study Sponsor Arbor Research Collaborative for Health
Collaborators National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Investigators
Study Chair: Saul Karpen, MD, PhD Children's Healthcare of Atlanta - Emory University
Study Director: Ed Doo, MD National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Principal Investigator: John Magee, MD University of Michigan Medical Center, Ann Arbor
Principal Investigator: Robert Merion, MD Arbor Research Collaborative of Health
Study Director: Averell Sherker, MD National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
PRS Account Arbor Research Collaborative for Health
Verification Date April 2020