We are updating the design of this site. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

PROBE: A Prospective Database of Infants With Cholestasis (PROBE)

This study is currently recruiting participants.
Verified September 2017 by Arbor Research Collaborative for Health
Sponsor:
ClinicalTrials.gov Identifier:
NCT00061828
First Posted: June 6, 2003
Last Update Posted: September 8, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by (Responsible Party):
Arbor Research Collaborative for Health
  Purpose
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.

Condition
Biliary Atresia Cholestasis

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 Arbor Research Collaborative for Health:

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. ]
    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)
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.

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.

  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   up to 6 Months   (Child)
Sexes Eligible for Study:   All
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.
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 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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00061828


Contacts
Contact: Peg Hill-Callahan, BS 734 369-9674 peg.hill-callahan@arborresearch.org
Contact: Joanne Lord, CCRC 734 369-9965 joanne.lord@arborresearch.org

Locations
United States, California
Children's Hospital Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Catherine Goodhue, CPNP    323-361-4566    cgoodhue@chla.usc.edu   
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: Michelle Hite    720-777-4690    michelle.hite@childrenscolorado.org   
Contact: Darci Anderson    720-777-9911    darci.anderson@childrenscolorado.org   
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: Rita Tory    404-785-1467    rita.tory@choa.org   
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    skelly@luriechildrens.org   
Contact: Mary Riordan, CCRP    312-227-4558    mriordan@luriechildrens.org   
Principal 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    clsawyer@iupui.edu   
Contact: Ann Klipsch, RN    317-944-9605    aeye@iupui.edu   
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
Saint Louis, Missouri, United States, 63110
United States, New York
Mount Sinai Medical Center Completed
New York, 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    julie.denlinger@cchmc.org   
Contact: Kelly Gindling    513-803-7482    kelly.gindling@cchmc.org   
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    erlichman@email.chop.edu   
Contact: Ashley Byrne    267-426-8613    byrneae@email.chop.edu   
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: Kathy Bukauskas    412-692-7703    kathryn.bukauskas@chp.edu   
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    tc2@bcm.edu   
Sub-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    ann.rutherford@hsc.utah.edu   
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    melissa.young@seattlechildrens.org   
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: Shannon Vandriel    416-813-7654 ext 201594    shannon.vandriel@sickkids.ca   
Sub-Investigator: Vicky Ng, MD         
Principal Investigator: Binita Kamath, MD         
Sponsors and Collaborators
Arbor Research Collaborative for Health
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Investigators
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 ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Arbor Research Collaborative for Health
ClinicalTrials.gov Identifier: NCT00061828     History of Changes
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 )
First Submitted: June 5, 2003
First Posted: June 6, 2003
Last Update Posted: September 8, 2017
Last Verified: September 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: The data will be transferred to NIDDK at the end of the study.

Additional relevant MeSH terms:
Cholestasis
Biliary Atresia
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Digestive System Abnormalities
Congenital Abnormalities