Biliary Atresia Study in Infants and Children (BASIC)

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2014 by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sponsor:
Information provided by (Responsible Party):
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
ClinicalTrials.gov Identifier:
NCT00345553
First received: June 27, 2006
Last updated: February 27, 2014
Last verified: February 2014
  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)

Resource links provided by NLM:


Further study details as provided by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK):

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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: June 2014
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
Study Population

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.

Criteria

Inclusion Criteria:

  1. 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).
  2. Subjects need to be greater than or equal to one year of age (with no upper age limit).
  3. Subject either have their native liver or have a confirmed liver transplantation.
  4. 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:

  1. Enrollment in the BARC study P003
  2. Inability to confirm original diagnostic evaluation of biliary atresia
  3. Inability or unwillingness of family or subject to participate in all scheduled visits.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00345553

Contacts
Contact: Ronald Sokol, MD 303-861-6669

Locations
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         
Sponsors and Collaborators
Investigators
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: February 27, 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
Digestive System Diseases
Digestive System Abnormalities
Congenital Abnormalities

ClinicalTrials.gov processed this record on August 18, 2014