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: | 1265 |
| Study Start Date: | May 2006 |
| Estimated Study Completion Date: | May 2019 |
| Estimated Primary Completion Date: | May 2019 (Final data collection date for primary outcome measure) |
| 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 LocationsPlease refer to this study by its ClinicalTrials.gov identifier: NCT00345553
| 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: 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 at San Francisco | Active, not recruiting |
| San Francisco, California, United States, 94143 | |
| United States, Colorado | |
| Children's Hospital Colorado | Recruiting |
| Aurora, Colorado, United States, 80045 | |
| Contact: Ronald Sokol, MD 720-777-6669 ronald.sokol@childrenscolorado.org | |
| Contact: Todd Miller 720-777-5304 todd.miller@childrenscolorado.org | |
| Sub-Investigator: Cara Mack, MD | |
| Sub-Investigator: Michael Narkewicz, MD | |
| Principal Investigator: Ronald Sokol, MD | |
| Sub-Investigator: Shikha Sundaram, MD | |
| United States, Georgia | |
| Children's Healthcare of Atlanta | Recruiting |
| Atlanta, Georgia, United States, 30322 | |
| Contact: Saul Karpen, MD, PhD 404-727-1463 saul.karpen@emory.edu | |
| 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 | |
| United States, Illinois | |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Recruiting |
| Chicago, Illinois, United States, 60614 | |
| Contact: Peter Whitington, MD 312-227-4616 pwhitington@luriechildrens.org | |
| Contact: Sue Kelly, RN BSN 312-227-3523 skelly@luriechildrens.org | |
| Sub-Investigator: Estella Alonso, MD | |
| Principal Investigator: Peter Whitington, MD | |
| Sub-Investigator: Lee Bass, MD | |
| United States, Indiana | |
| Riley Children's Hospital | Recruiting |
| Indianapolis, Indiana, United States, 46202 | |
| Contact: Jean Molleston, MD 317-944-3774 jpmolles@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 |
| St 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: Jorge Bezerra, MD 513-636-4928 jorge.bezerra@cchmc.org | |
| Contact: Julie Denlinger 513-636-7818 julie.denlinger@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: Kathy Loomes, MD 215-426-7223 loomes@email.chop.edu | |
| Contact: Jessi Erlichman 215-590-2525 erlichman@email.chop.edu | |
| Principal Investigator: Kathy Loomes, MD | |
| Sub-Investigator: Elizabeth Rand, MD | |
| Sub-Investigator: David Piccoli, MD | |
| Children's Hospital at Pittsburgh | Recruiting |
| Pittsburgh, Pennsylvania, United States, 15213 | |
| Contact: Benjamin Shneider, MD 412-692-5180 Benjamin.Shneider@chp.edu | |
| Contact: Kathy Bukauskas 412-692-7703 kathryn.bukauskas@chp.edu | |
| Sub-Investigator: David Perlmutter, MD | |
| Sub-Investigator: Robert Squires, MD | |
| Principal Investigator: Benjamin Shneider, MD | |
| Sub-Investigator: Veena Venkat, 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 206-987-2775 karen.murray@seattlechildrens.org | |
| 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 | |
| Hospital for Sick Children | Recruiting |
| Toronto, Ontario, Canada, M5G 1X8 | |
| Contact: Binita Kamath, MD 416-813-7654 ext 28193 binita.kamath@sickkids.ca | |
| Contact: Kelsey Hunt 416-813-7654 ext 201594 kelsey.hunt@sickkids.ca | |
| Sub-Investigator: Vicki Ng, MD | |
| Principal Investigator: Binita Kamath, 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 by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| 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: | September 15, 2014 |
| 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 |
Congenital Abnormalities Digestive System Abnormalities Digestive System Diseases |
ClinicalTrials.gov processed this record on March 03, 2015