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 : April 22, 2022
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Condition or disease |
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Biliary Atresia |
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.
Study Type : | Observational |
Estimated Enrollment : | 1265 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | Biliary Atresia Study in Infants and Children (BASIC) |
Actual Study Start Date : | May 16, 2006 |
Estimated Primary Completion Date : | May 2024 |
Estimated Study Completion Date : | May 2024 |

Group/Cohort |
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1
Biliary atresia subjects who have their native liver
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2
Biliary atresia subjects who have had a liver transplant
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- 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 etiology is a result of a genetic defect and the second is to examine the influence of genetics on disease acquisition.
- To identify polymorphisms that may be important in disease progression such as Human leukocyte antigen (HLA) polymorphisms [ Time Frame: Specimens for this aim are collected once during study, usually at baseline. ]
- 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

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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 |
Inclusion Criteria:
- 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).
- 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).
- Participants either have their native liver or have a confirmed liver transplantation.
- 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:
- Currently participating in the ChiLDReN study PROBE
- Inability to confirm original diagnostic evaluation of biliary atresia
- Inability or unwillingness of family or participant to participate in all scheduled visits.

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
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 |
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 | |
Sub-Investigator: Nisreen Soufi, MD | |
Sub-Investigator: Rohit Kohil, 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: Matthew Steinbeiss 720-777-4800 matthew.steinbeiss@childrenscolorado.org | |
Contact: Mikaela Kauma 720-777-1294 mikaela.kauma@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: Cindy Sawyers, BSRT 317-944-1421 clsawyer@iu.edu | |
Contact: Ann Klipsch, RN 317-274-9605 aeye@iu.edu | |
Principal Investigator: Jean Molleston, MD | |
Sub-Investigator: Molly Bozic, MD | |
Sub-Investigator: Girish Rao, 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 | |
Sub-Investigator: Jorge Bezerra, MD | |
Sub-Investigator: James Heubi, MD | |
Principal 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 | |
Contact: Adam Kufen, RN, CCRC 412-692-6558 adam.kufen@chp.org | |
Sub-Investigator: Robert Squires, MD | |
Sub-Investigator: James Squires, MD | |
Principal 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 | |
Sub-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 | |
Contact: Tyler Hall 801-587-5670 tyler.hall@hsc.utah.edu | |
Sub-Investigator: Stephen Guthery, MD | |
Principal Investigator: Kyle Jensen, MD | |
Sub-Investigator: Linda Book, MD | |
United States, Washington | |
Seattle Children's Hospital | Recruiting |
Seattle, Washington, United States, 98105 | |
Contact: Melissa Young 206-987-1037 melissa.young@seattlechildrens.org | |
Contact: Kara Cooper 206-987-4636 kara.cooper@seattlechildrens.org | |
Principal Investigator: Simon Horslen, MD | |
Sub-Investigator: Evelyn Hsu, MD | |
Sub-Investigator: Niviann Blondet, 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: Claudia Quammie 416-813-7654 ext 201594 claudia.quammie@sickkids.ca | |
Sub-Investigator: Vicki Ng, MD | |
Principal Investigator: Binita Kamath, MD |
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) |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Biliary Atresia Cholestasis |
Biliary Atresia Bile Duct Diseases Biliary Tract Diseases |
Digestive System Diseases Digestive System Abnormalities Congenital Abnormalities |