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Biliary Atresia Study in Infants and Children (BASIC)

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ClinicalTrials.gov Identifier: NCT00345553
Recruitment Status : Recruiting
First Posted : June 28, 2006
Last Update Posted : February 22, 2019
Sponsor:
Collaborator:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Information provided by (Responsible Party):
Arbor Research Collaborative for Health

Brief Summary:
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 or disease
Biliary Atresia

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 Childhood Liver Disease Research Network (ChiLDReN), a National Institute of Diabetes & Digestive and Kidney Diseases (NIDDK) funded network.


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Study Type : Observational
Estimated Enrollment : 1265 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Biliary Atresia Study in Infants and Children (BASIC)
Study Start Date : May 2006
Estimated Primary Completion Date : May 2019
Estimated Study Completion Date : May 2019

Resource links provided by the National Library of Medicine


Group/Cohort
1
Biliary atresia subjects who have their native liver
2
Biliary atresia subjects who have had a liver transplant



Primary Outcome Measures :
  1. 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. ]
    The genetics of BA may be investigated on two levels. The first is to identify a group of patients whose eitiology is a result of a genetic defect and the second is to examine the influence of genetics on disease acquisition.


Secondary Outcome Measures :
  1. 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. ]
  2. 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. ]
    Understanding the natural history of a disease is a prerequisite to interpreting disease severity, identifying patterns of illness, identifying early predictors of outcome and understanding the advantages or trade-offs of therapeutic interventions.


Biospecimen Retention:   Samples With DNA
Samples of blood will be collected for research purposes.


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Recruitment will be open to any eligible subject. The method of contacting the study subjects will conform to local IRB guidelines, but in general: the parents or guardians of all eligible subjects at each ChiLDReN center, or the subjects themselves if 18 years of age or older, will be approached to participate. New patients who are not participating in ChiLDReN study PROBE may be approached for study participation, but will not be eligible for enrollment until 6 months of age.
Criteria

Inclusion Criteria:

  1. Participants 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. Participants need to be >6 months of age up to and equal to the age of 20 (participants enrolled at 20 years of age will have one visit).
  3. Participants either have their native liver or have a confirmed liver transplantation.
  4. Parent, guardian or participant (if 18 years of age or older) is willing to provide informed consent and, when appropriate, the participant is willing to assent.

Exclusion Criteria:

  1. Currently participating in the ChiLDReN study PROBE
  2. Inability to confirm original diagnostic evaluation of biliary atresia
  3. Inability or unwillingness of family or participant to participate in all scheduled visits.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00345553


Contacts
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Contact: Joanne Lord, LPN,BA,CCRC 734-369-9965 joanne.lord@arborresearch.org
Contact: Terese A Howell, BS, CCRC 734-369-9683 terri.howell@arborresearch.org

Locations
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United States, California
Children's Hospital of Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Catherine Goodhue, CPNP    323-361-4566    cgoodhue@chla.usc.edu   
Principal Investigator: Kasper Wang, 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: Michelle Hite    720-777-4690    michelle.hite@childrenscolorado.org   
Contact: Matthew Steinbeiss    720-777-4800    matthew.steinbeiss@childrenscolorado.org   
Principal Investigator: Ronald J Sokol, MD         
Sub-Investigator: Michael Narkewicz, MD         
Sub-Investigator: Cara Mack, MD         
Sub-Investigator: Shikha Sundaram, MD         
Sub-Investigator: Amy Feldman, MD         
Sub-Investigator: Dania Brigham, MD         
United States, Georgia
Children's Healthcare of Atlanta - Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Jeanette McFall    404-785-0421    jeanette.mcfall@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: Sue Kelly, RN, BSN    312-227-3523    skelly@luriechildrens.org   
Contact: Mary Riordan, CCRP    312-227-4558    mriordan@luriechildrens.org   
Principal Investigator: Estella Alonso, MD         
Sub-Investigator: Lee Bass, MD         
United States, Indiana
Riley Children's Hospital Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Ann Klipsch, RN    317-944-9605    aeye@iu.edu   
Contact: Cindy Sawyers, RT    317-944-1421    clsawyer@iu.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
Saint 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: 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: Jessi Erlichman    215-590-2525    erlichman@email.chop.edu   
Contact: Iraklis Petrof    267-426-8613    petrofi@email.chop.edu   
Principal Investigator: Kathy Loomes, MD         
Sub-Investigator: Elizabeth Rand, MD         
Sub-Investigator: David Piccoli, MD         
UPMC Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Kathryn Bukauskas, RN, CCRC    412-692-7703    kathryn.bukauskas@chp.edu   
Principal Investigator: Robert Squires, MD         
Sub-Investigator: Veena Venkat, MD         
Sub-Investigator: James Squires, MD         
Sub-Investigator: Patrick Mckiernan, MD         
United States, Texas
Texas Children's Hospital/Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Laurel Cavallo    832-822-1053    laurel.cavallo@bcm.edu   
Contact: Cynthia Tsai    832-822-3634    ct2@bcm.edu   
Principal Investigator: Paula Hertel, MD         
Principal Investigator: Benjamin Shneider, MD         
United States, Utah
University of Utah Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Ann Rutherford    801-585-9495    ann.rutherford@hsc.utah.edu   
Sub-Investigator: Stephen Guthery, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Melissa Young    206-987-1037    melissa.young@seattlechildrens.org   
Principal Investigator: Karen Murray, MD         
Sub-Investigator: Simon Horslen, MD         
Sub-Investigator: Evelyn Hsu, MD         
Canada, Ontario
Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Deepika Sharma    416-813-7654 ext 201594    Deepika.sharma@sickkids.ca   
Contact: Elena Reydman    416-813-7654    elena.reydman@sickkids.ca   
Sub-Investigator: Vicki Ng, MD         
Principal Investigator: Binita Kamath, MD         
Sponsors and Collaborators
Arbor Research Collaborative for Health
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Investigators
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Study Chair: Vicky Ng, MD The Hospital for Sick Children, Toronto, ON, Canada
Study Director: Ed Doo, MD National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Principal Investigator: John C Magee, MD University of Michigan
Principal Investigator: Robert M Merion, MD Arbor Research Collaborative for Health - Data Coordinating Center
Study Director: Averell Sherker, MD National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Arbor Research Collaborative for Health
ClinicalTrials.gov Identifier: NCT00345553     History of Changes
Other Study ID Numbers: BASIC Study - ChiLDReN Network
U01DK103149 ( U.S. NIH Grant/Contract )
U01DK103140 ( U.S. NIH Grant/Contract )
U01DK103135 ( U.S. NIH Grant/Contract )
U01DK084575 ( U.S. NIH Grant/Contract )
U01DK084538 ( U.S. NIH Grant/Contract )
U01DK084536 ( U.S. NIH Grant/Contract )
U01DK062503 ( U.S. NIH Grant/Contract )
U01DK062500 ( U.S. NIH Grant/Contract )
U01DK062497 ( U.S. NIH Grant/Contract )
U01DK062481 ( U.S. NIH Grant/Contract )
U01DK062470 ( U.S. NIH Grant/Contract )
U01DK062466 ( U.S. NIH Grant/Contract )
U01DK062456 ( U.S. NIH Grant/Contract )
U01DK062453 ( U.S. NIH Grant/Contract )
U01DK062452 ( U.S. NIH Grant/Contract )
U01DK062445 ( U.S. NIH Grant/Contract )
U01DK062436 ( U.S. NIH Grant/Contract )
First Posted: June 28, 2006    Key Record Dates
Last Update Posted: February 22, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: The data will be transferred to NIDDK at the end of the study.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Arbor Research Collaborative for Health:
Biliary Atresia
Cholestasis

Additional relevant MeSH terms:
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Biliary Atresia
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Digestive System Abnormalities
Congenital Abnormalities