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| Sponsored by: |
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
|---|---|
| Information provided by: | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
| ClinicalTrials.gov Identifier: | NCT00345553 |
Purpose
Little is known about the factors that cause biliary atresia nor the factors that influence disease progression. The purpose of this study is to collect the pertinent clinical information, genetic material and body fluid samples to enable investigators to address the following aims: To identify the gene or genes implicated in the etiology of BA; To identify polymorphisms that may be important in disease progression such as HLA polymorphisms; To characterize the natural history of the older, non-transplanted child with BA.
| Condition |
|---|
|
Biliary Atresia |
| Study Type: | Observational |
| Study Design: | Cohort, Prospective |
| Official Title: | Biliary Atresia Study in Infants and Children (BASIC) |
Samples of blood and urine will be collected for research purposes.
| Estimated Enrollment: | 500 |
| Study Start Date: | May 2006 |
| Estimated Study Completion Date: | June 2012 |
| Groups/Cohorts |
|---|
|
1
Biliary atresia subjects who have their native liver
|
|
2
Biliary atresia subjects who have had a liver transplant
|
Little is known about the factors that cause biliary atresia nor the factors that influence disease progression. A variety of genetic, autoimmune and environmental influences have been hypothesized to be important. Most studies to date have focused on the neonate and young child with BA, yet the older surviving child with BA can provide important information about genetics, as well as, natural history.
The purpose of this study is to collect the pertinent clinical information, genetic material and body fluid samples to enable investigators to address the following hypotheses:
Hypothesis 1: A genetic defect is a likely causative factor for BA among children with BA and multiple congenital anomalies.
Hypothesis 2: Autoimmune factors are likely to contribute to disease progression or acquisition and can be identified by correlating HLA among children with BA to healthy controls and by comparison of those who develop early complications including, variceal bleed, ascites, and growth failure compared to those who do not.
Hypothesis 3a: Sentinel events such as variceal bleeding, ascites and growth failure are earlier predictors of death or need for liver transplantation than the pediatric end-stage liver disease score (PELD) Hypothesis 3b: Health related quality of life will be impaired compared to healthy age matched children and relate to severity of illness.
Hypothesis 3c: Growth failure as measured by anthropometrics and nutritional supplementation will be predictive of onset of sentinel events (ascites, variceal bleed, death, and transplant) in the following 24 months.
This study will be performed by the Biliary Atresia Research Clinical Research Consortium (BARC), an NIDDK-funded network.
Eligibility| Ages Eligible for Study: | 1 Year to 75 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
The parents or guardians of all eligible subjects at each BARC center, or the subjects themselves if 18 years of age or older, will receive a letter of introduction, followed by a telephone call and, if willing, arrangement of an appointment at which time informed consent will be obtained. New patients who are at least one year of age and not participating in the BARC PROBE study will also be approached.
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Ronald Sokol, MD | 303-861-6669 |
| United States, California | |
| University of California at San Francisco | Recruiting |
| San Francisco, California, United States, 94143 | |
| Contact: Philip Rosenthal, MD 415-476-5892 prosenth@peds.ucsf.edu | |
| Principal Investigator: Philip Rosenthal, MD | |
| United States, Colorado | |
| The Children's Hospital | Recruiting |
| Denver, Colorado, United States, 80218 | |
| Contact: Ronald Sokol, MD 303-861-6669 sokol.ronald@tchden.edu | |
| Sub-Investigator: Cara Mack, MD | |
| Sub-Investigator: Michael Narkewicz, MD | |
| Principal Investigator: Ronald Sokol, MD | |
| United States, Illinois | |
| Children's Memorial Hospital | Recruiting |
| Chicago, Illinois, United States, 60614 | |
| Contact: Peter Whitington, MD 773-880-4643 p-whitington@northwestern.edu | |
| Sub-Investigator: Riccardo Superina, MD | |
| Principal Investigator: Peter Whitington, MD | |
| United States, Maryland | |
| Johns Hopkins School of Medicine | Recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Contact: Kathleen Schwarz, MD 410-955-8769 kschwarz@jhmi.edu | |
| Principal Investigator: Kathleen Schwarz, MD | |
| United States, Missouri | |
| Washington University School of Medicine | Recruiting |
| St Louis, Missouri, United States, 63110 | |
| Contact: Ross Shepherd, MD 314-454-2337 shepherd_r@kids.wustl.edu | |
| Principal Investigator: Ross Shepherd, MD | |
| United States, New York | |
| Mount Sinai Medical Center | Recruiting |
| New York, New York, United States, 10029 | |
| Contact: Frederick J Suchy, MD 212-241-6933 frederick.suchy@mssm.edu | |
| Principal Investigator: Frederick J Suchy, MD | |
| Sub-Investigator: Nanda Kerkar, MD | |
| United States, Ohio | |
| Children's Hospital Medical Center | Recruiting |
| Cincinnati, Ohio, United States, 45229 | |
| Contact: Jorge Bezerra, MD 513-636-4928 jorge.bezerra@chmcc.org | |
| Principal Investigator: Jorge Bezerra, MD | |
| Sub-Investigator: John Bucuvalas, MD | |
| United States, Pennsylvania | |
| Children's Hospital at Pittsburgh | Recruiting |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| Contact: Benjamin Shneider, MD 412-692-5412 Benjamin.Shneider@chp.edu | |
| Sub-Investigator: David Perlmutter, MD | |
| Sub-Investigator: Robert Squires, MD | |
| Principal Investigator: Benjamin Shneider, MD | |
| Children's Hospital of Philadelphia | Recruiting |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Contact: Barbara Haber, MD 215-590-2985 haber@email.chop.edu | |
| Principal Investigator: Barbara Haber, MD | |
| Sub-Investigator: Elizabeth Rand, MD | |
| United States, Texas | |
| Texas Children's Hospital/Baylor College of Medicine | Recruiting |
| Houston, Texas, United States, 77030 | |
| Contact: Saul J Karpen, MD 832-824-3754 skarpen@bcm.tmc.edu | |
| Principal Investigator: Saul J Karpen, MD | |
| Study Chair: | Ronald Sokol, MD | The Children's Hospital, Denver |
| Study Director: | Patricia Robuck, Phd | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
More Information
| Responsible Party: | University of Michigan Medical Center ( John Magee, MD, Principal Investigator ) |
| Study ID Numbers: | BASIC |
| Study First Received: | June 27, 2006 |
| Last Updated: | January 5, 2009 |
| ClinicalTrials.gov Identifier: | NCT00345553 |
| Health Authority: | United States: Federal Government |
|
Biliary Atresia Cholestasis |
|
Digestive System Abnormalities Digestive System Diseases Bile Duct Diseases Cholestasis |
Biliary Tract Diseases Biliary atresia Congenital Abnormalities Biliary Atresia |
