Biliary Atresia Study in Infants and Children (BASIC)
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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: | Observational Model: Cohort Time Perspective: Prospective |
| Official Title: | Biliary Atresia Study in Infants and Children (BASIC) |
- To identify the gene or genes implicated in the etiology of BA [ Time Frame: Specimens for this aim are collected once during study, usually at baseline. ] [ Designated as safety issue: No ]
- To identify polymorphisms that may be important in disease progression such as HLA polymorphisms [ Time Frame: Specimens for this aim are collected once during study, usually at baseline. ] [ Designated as safety issue: No ]
- Define the natural history of the older, non-transplanted child with biliary atresia [ Time Frame: Observational information collected at entrance into study as well as at each yearly follow-up visit. ] [ Designated as safety issue: No ]
Biospecimen Retention: Samples With DNA
Samples of blood and urine will be collected for research purposes.
| Estimated Enrollment: | 1200 |
| Study Start Date: | May 2006 |
| Estimated Study Completion Date: | December 2012 |
| Groups/Cohorts |
|---|
|
1
Biliary atresia subjects who have their native liver
|
|
2
Biliary atresia subjects who have had a liver transplant
|
Detailed Description:
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:
- Subjects need to have a confirmed diagnosis of BA determined by chart review including review of pertinent diagnostic biopsy reports, radiologic reports and surgical reports (if surgery was performed).
- Subjects need to be greater than or equal to one year of age (with no upper age limit).
- Subject either have their native liver or have a confirmed liver transplantation.
- Parent, guardian or subject (if 18 years of age or older) is willing to provide informed consent and, when appropriate, the subject is willing to assent.
Exclusion Criteria:
- Enrollment in the BARC study P003
- Inability to confirm original diagnostic evaluation of biliary atresia
- Inability or unwillingness of family or subject to participate in all scheduled visits.
Contacts and Locations| Contact: Ronald Sokol, MD | 303-861-6669 |
| United States, California | |
| Children's Hospital of Los Angeles | Recruiting |
| Los Angeles, California, United States, 90027 | |
| Contact: Kasper Wang, MD 323-361-2338 kwang@chla.usc.edu | |
| Contact: Cat Goodhue, CPNP 323-361-4566 cgoodhue@chla.usc.edu | |
| Principal Investigator: Kasper Wang, MD | |
| 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, Georgia | |
| Children's Healthcare of Atlanta | Recruiting |
| Atlanta, Georgia, United States, 30322 | |
| Contact: Saul Karpen, MD, PhD 404-727-1463 skarpen@emory.edu | |
| Principal Investigator: Saul Karpen, MD PhD | |
| 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, Indiana | |
| Riley Children's Hospital | Recruiting |
| Indianapolis, Indiana, United States, 46202 | |
| Contact: Jean Molleston, MD 317-944-3774 jpmolles@iupui.edu | |
| Principal Investigator: Jean Molleston, 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: Yumi Turmelle, MD 314-454-6173 turmelle_y@kids.wustl.edu | |
| Principal Investigator: Yumi Turmelle, MD | |
| United States, New York | |
| Mount Sinai Medical Center | Recruiting |
| New York, New York, United States, 10029 | |
| Contact: Nanda Kerkar, MD 212-659-8060 nanda.kerkar@msnyuhealth.org | |
| Contact: Mariel Boyd 212-659-8046 mariel.boyd@mountsinai.org | |
| Sub-Investigator: Ronen Arnon, 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 of Philadelphia | Recruiting |
| Philadelphia, Pennsylvania, United States, 19104 | |
| Contact: Kathy Loomes, MD 215-426-7223 loomes@email.chop.edu | |
| Principal Investigator: Kathy Loomes, MD | |
| Sub-Investigator: Elizabeth Rand, MD | |
| 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 | |
| United States, Texas | |
| Texas Children's Hospital/Baylor College of Medicine | Active, not recruiting |
| Houston, Texas, United States, 77030 | |
| United States, Washington | |
| Childern's Hospital & Regional Medical Center | Recruiting |
| Seattle, Washington, United States, 98105 | |
| Contact: Karen Murray, MD 206-987-2587 karen.murray@seattlechildrens.org | |
| Principal Investigator: Karen Murray, MD | |
| Canada, Ontario | |
| Hospital for Sick Children | Recruiting |
| Toronto, Ontario, Canada, M5G 1X8 | |
| Contact: Vicki Ng, MD 416-813-6263 vicky.ng@sickkids.ca | |
| Principal Investigator: Vicki Ng, MD | |
| Study Chair: | Ronald Sokol, MD | Children's Hospital Colorado |
| Study Director: | Patricia Robuck, Phd | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
More Information
Additional Information:
No publications provided
| Responsible Party: | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
| ClinicalTrials.gov Identifier: | NCT00345553 History of Changes |
| Other Study ID Numbers: | BASIC |
| Study First Received: | June 27, 2006 |
| Last Updated: | August 27, 2012 |
| Health Authority: | United States: Federal Government |
Keywords provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):
|
Biliary Atresia Cholestasis |
Additional relevant MeSH terms:
|
Biliary Atresia Bile Duct Diseases Biliary Tract Diseases |
Digestive System Diseases Digestive System Abnormalities Congenital Abnormalities |
ClinicalTrials.gov processed this record on May 23, 2013