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Trial record 15 of 90 for:    Open Studies | "Cholestasis"

Biliary Atresia Research Consortium (PROBE)

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 2016 by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by (Responsible Party):
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Identifier:
First received: June 5, 2003
Last updated: September 28, 2016
Last verified: September 2016
Biliary atresia and idiopathic neonatal hepatitis are the most common causes of jaundice and hyperbilirubinemia that continue beyond the newborn period. The long term goal of the Biliary Atresia Research Consortium (BARC) is to establish a database of clinical information and serum and tissue samples from children with biliary atresia (BA) and idiopathic neonatal hepatitis (INH) to facilitate research and to perform clinical, epidemiological and therapeutic trials in these two important pediatric liver diseases.

Biliary Atresia

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Childhood Liver Disease Research and Education Network (CHILDREN): A Prospective Database of Infants With Cholestasis

Resource links provided by NLM:

Further study details as provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):

Primary Outcome Measures:
  • 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. ] [ Designated as safety issue: No ]
    disease progression defined by transplant date, date of death, worsening liver function, and complications related to worsening liver function

Biospecimen Retention:   Samples With DNA
Samples of blood, urine and liver tissue samples will be collected for research purposes.

Estimated Enrollment: 1000
Study Start Date: May 2004
Estimated Study Completion Date: May 2019
Estimated Primary Completion Date: May 2019 (Final data collection date for primary outcome measure)
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.

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 and neonatal hepatitis, in order to perform research in these important liver problems. Children will be screened and enrolled at presentation at the participating pediatric liver sites. Subjects diagnosed with biliary atresia will be followed intensively for the first year, at 18 months of age, and then annually up to 15 years of age. Other subjects 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 biopsy 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 and urine 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, biliary remnant and bile specimens, if 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 subject's condition. As part of the standard of care, the study will follow-up and record progress of the liver problem by routine clinical examinations and laboratory tests for up to 15 years. All data from this study will be kept in a secure research database at the data coordinating center.


Ages Eligible for Study:   up to 6 Months   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
This study population will be selected from the patient base of participating speciality care clinics.


  • 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.


  • 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 TPN-associated cholestasis (except when diagnosed with biliary atresia or another cholestatic disease being studied by ChiLDREN).
  • Diagnosis with ECMO-associated cholestasis.
  • Birth weight less than 1500g (except when diagnosed with biliary atresia).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00061828

Contact: Peg Hill-Callahan, BS 734 369-9674
Contact: Terese A. Howell, BS, CCRC 734 369-9683

United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Catherine Goodhue, CPNP    323-361-4566   
Principal Investigator: Kasper Wang, MD         
Sub-Investigator: Nanda Kerker, MD         
Sub-Investigator: Sonia Michail, MD         
Sub-Investigator: Danny Thomas, MD         
University of California Active, not recruiting
San Francisco, California, United States, 94143
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Todd Miller    720-777-5304   
Contact: Michelle Hite    720-777-4690   
Principal Investigator: Ronald Sokol, MD         
Sub-Investigator: Cara Mack, MD         
Sub-Investigator: Michael Narkewicz, MD         
Sub-Investigator: Shikha Sundaram, MD         
United States, Georgia
Children's Healthcare of Atlanta (Emory University) Recruiting
Atlanta, Georgia, United States, 30322
Contact: Dana Hankerson-Dyson    404-785-6027   
Contact: Rita Tory    404-785-1467   
Principal Investigator: Saul Karpen, MD, PhD         
Sub-Investigator: Nitika Gupta, MD         
Sub-Investigator: Rene Romero, MD         
Sub-Investigator: Miriam Vos, MD MSPH         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60614
Contact: Sue Kelly, RN, BSN    312-227-3523   
Contact: Elizabeth Kaurs    312-227-4558   
Principal Investigator: Peter Whitington, MD         
Sub-Investigator: Estella Alonso, MD         
Sub-Investigator: Lee Bass, MD         
United States, Indiana
Riley Hospital for Children Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Cindy Sawyers, BSRT    317-944-1421   
Contact: Ann Klipsch, RN    317-944-9605   
Principal Investigator: Jean Molleston, MD         
Sub-Investigator: Molly Bozic, MD         
United States, Maryland
Johns Hopkins School of Medicine Completed
Baltimore, Maryland, United States, 21287
United States, Missouri
Washington University School of Medicine Completed
St Louis, Missouri, United States, 63110
United States, New York
Mount Sinai Medical Center Completed
New York City, New York, United States, 10029
United States, Ohio
Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Julie Denlinger    513-636-7818   
Contact: Andrea Ferris    5138030675   
Principal Investigator: Jorge Bezerra, MD         
Sub-Investigator: James Heubi, MD         
Sub-Investigator: Alexander Miethke, MD         
Sub-Investigator: Joseph Palermo, MD         
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Jessi Erlichman, MPH    215-590-2525   
Contact: Ashley Byrne    267-426-8613   
Principal Investigator: Kathleen Loomes, MD         
Sub-Investigator: Elizabeth Rand, MD         
Sub-Investigator: David Piccoli, MD         
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Madeline Schulte    412-692-5811   
Contact: Kathy Bukauskas, RN    412-692-7703   
Principal Investigator: Robert Squires, Jr, MD         
Sub-Investigator: Veena Venkat, MD         
United States, Texas
Baylor College of Medicine Recruiting
Houston,, Texas, United States, 77030
Contact: Cynthia Tsai, MPH    832-822-3634   
Principal Investigator: Paula Hertel, MD         
Principal Investigator: Benjamin Shneider, MD         
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Ann Rutherford    801-585-9495   
Contact: Kyle Berg    801-587-5670   
Principal Investigator: Stephen Guthery, MD         
Sub-Investigator: Kyle Jensen, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Melissa Young    206-987-1037   
Principal Investigator: Karen Murray, MD         
Sub-Investigator: Simon Horslen, MD         
Sub-Investigator: Evelyn Shu, MD         
Canada, Ontario
The Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Claudia Quammie    416-813-7654 ext 201594   
Sub-Investigator: Vicky Ng, MD         
Principal Investigator: Binita Kamath, MD         
Sponsors and Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Study Chair: Ronald Sokol, MD Children's Hospital Colorado
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)
  More Information

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):

Responsible Party: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Identifier: NCT00061828     History of Changes
Other Study ID Numbers: BARC - IND 
Study First Received: June 5, 2003
Last Updated: September 28, 2016
Health Authority: United States: Federal Government
Individual Participant Data  
Plan to Share IPD: Yes
Plan Description: The data will be transferred to NIDDK at the end of the study.

Additional relevant MeSH terms:
Biliary Atresia
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Digestive System Abnormalities
Congenital Abnormalities processed this record on October 21, 2016