Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
Trial record 15 of 66 for:    Open Studies | Exclude Unknown | digestive disease | NIDDK

Biliary Atresia Study in Infants and Children (BASIC)

This study is currently recruiting participants. (see Contacts and Locations)
Verified September 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: September 15, 2014
Last verified: September 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: 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
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: 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         
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 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 November 25, 2014