A Prospective Database of Infants With Cholestasis (PROBE)
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| ClinicalTrials.gov Identifier: NCT00061828 |
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Recruitment Status :
Suspended
(Study visits suspended due to COVID-19 pandemic.)
First Posted : June 6, 2003
Last Update Posted : April 2, 2020
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| Condition or disease |
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| Biliary Atresia Neonatal Cholestasis |
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 |
| Estimated Enrollment : | 1000 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Official Title: | Childhood Liver Disease Research Network (ChiLDReN): A Prospective Database of Infants With Cholestasis |
| Study Start Date : | May 2004 |
| Estimated Primary Completion Date : | May 2024 |
| Estimated Study Completion Date : | May 2024 |
| Group/Cohort |
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Biliary Atresia
Infants presenting with cholestasis who are diagnosed with biliary atresia.
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Non-Biliary Atresia
Infants presenting with cholestasis without a diagnosis of biliary atresia.
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- 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
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| Ages Eligible for Study: | up to 6 Months (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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).
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
| United States, California | |
| Children's Hospital Los Angeles | |
| Los Angeles, California, United States, 90027 | |
| University of California | |
| San Francisco, California, United States, 94143 | |
| United States, Colorado | |
| Children's Hospital Colorado | |
| Aurora, Colorado, United States, 80045 | |
| United States, Georgia | |
| Children's Healthcare of Atlanta - Emory University | |
| Atlanta, Georgia, United States, 30322 | |
| United States, Illinois | |
| Ann & Robert H. Lurie Children's Hospital of Chicago | |
| Chicago, Illinois, United States, 60614 | |
| United States, Indiana | |
| Riley Hospital for Children | |
| Indianapolis, Indiana, United States, 46202 | |
| United States, Maryland | |
| Johns Hopkins School of Medicine | |
| Baltimore, Maryland, United States, 21287 | |
| United States, Missouri | |
| Washington University School of Medicine | |
| Saint Louis, Missouri, United States, 63110 | |
| United States, New York | |
| Mount Sinai Medical Center | |
| New York, New York, United States, 10029 | |
| United States, Ohio | |
| Cincinnati Children's Hospital Medical Center | |
| Cincinnati, Ohio, United States, 45229 | |
| United States, Pennsylvania | |
| Children's Hospital of Philadelphia | |
| Philadelphia, Pennsylvania, United States, 19104 | |
| UPMC Children's Hospital of Pittsburgh | |
| Pittsburgh, Pennsylvania, United States, 15224 | |
| United States, Texas | |
| Baylor College of Medicine | |
| Houston, Texas, United States, 77030 | |
| United States, Utah | |
| University of Utah | |
| Salt Lake City, Utah, United States, 84113 | |
| United States, Washington | |
| Seattle Children's Hospital | |
| Seattle, Washington, United States, 98105 | |
| Canada, Ontario | |
| The Hospital for Sick Children | |
| Toronto, Ontario, Canada, M5G 1X8 | |
| 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) |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Cholestasis Biliary Atresia Bile Duct Diseases Biliary Tract Diseases |
Digestive System Diseases Digestive System Abnormalities Congenital Abnormalities |