Biliary Atresia Study in Infants and Children - Full Text View

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:

Genetic and Rare Diseases Information Center resources: Biliary Atresia

U.S. FDA Resources

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:	6 Months and older (Child, Adult, Senior)
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:

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 (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 PROBE study
Inability to confirm original diagnostic evaluation of biliary atresia
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: Peg Hill-Callahan, BS, LSW	734-369-9674	peg.hill-callahan@arborresearch.org
Contact: Terese Howell, BS	734 369-9683	thowell@umich.edu

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: Robert Squires, MD 412-692-5180 robert.squires@chp.edu
Contact: Kathy Bukauskas 412-692-7703 kathryn.bukauskas@chp.edu
Sub-Investigator: David Perlmutter, MD
Principal Investigator: Robert Squires, MD
Sub-Investigator: Veena Venkat, MD
Sub-Investigator: Jim Squires, MD
United States, Texas
Texas Children's Hospital/Baylor College of Medicine	Recruiting
Houston, Texas, United States, 77030
Contact: Paula Hertel, MD 832-824-2099 phertel@bcm.edu
Contact: Cynthia Tsai 832- 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: Kyle Jensen, MD 801-662-2900 kyle.jensen@hsc.utah.edu
Contact: Ann Rutherford 801-585-9495 ann.rutherford@hsc.utah.edu
Sub-Investigator: Stephen Guthery, MD
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: Claudia Quammie 416-813-7654 ext 201594 claudia.quammie@sickkids.ca
Sub-Investigator: Vicki Ng, MD
Principal Investigator: Binita Kamath, MD

Sponsors and Collaborators

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Investigators


Study Chair:	Ronald Sokol, MD	Children's Hospital Colorado
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)

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Ng VL, Haber BH, Magee JC, Miethke A, Murray KF, Michail S, Karpen SJ, Kerkar N, Molleston JP, Romero R, Rosenthal P, Schwarz KB, Shneider BL, Turmelle YP, Alonso EM, Sherker AH, Sokol RJ; Childhood Liver Disease Research and Education Network (CHiLDREN). Medical status of 219 children with biliary atresia surviving long-term with their native livers: results from a North American multicenter consortium. J Pediatr. 2014 Sep;165(3):539-546.e2. doi: 10.1016/j.jpeds.2014.05.038. Epub 2014 Jul 9. Erratum in: J Pediatr. 2015 Jan;166(1):211.


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 20, 2016
Health Authority:	United States: Federal Government
Individual Participant Data
Plan to Share IPD:	Yes
Plan Description:	The data will be transferred to NIDDK at the end of the study.

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 September 29, 2016

Biliary Atresia Study in Infants and Children (BASIC)