Biliary AtresiaBASICNCT00345553
Biliary Atresia Study in Infants and Children
An observational study of Biliary Atresia, run by Arbor Research Collaborative for Health at 16 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 months to 20 years
- Conditions the study is for
- Biliary Atresia
- How many people
- 1,265 participants
- Where
- 16 sitesin 2 countries
- Study dates
- 16 May 2006to 31 May 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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 with their native liver.
- 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.
Who cannot join
- 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.
- History of liver transplantation.
Trial locations and contacts
16 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
16 sites in 2 countries
United States15 sites, 9 recruiting
- Recruiting
Children's Healthcare of Atlanta - Emory University
Atlanta, Georgia, 30322
Katelynn Harris, 404-785-0421, katelynn.harris@choa.org
Jordyn Turner, 404-785-3690, jordyn.turner@choa.org
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Kelly Gilmartin, 720-777-2961, kelly.gilmartin@childrenscolorado.org
Michelle Landis, 720-777-8884, michelle.landis@childrenscolorado.org
- Completed
Johns Hopkins School of Medicine
Baltimore, Maryland, 21287
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60614
Jacqueline Alexander, 312-227-3523, jaalexander@luriechildrens.org
Angela Anthony, 312-227-4559, aanthony@luriechildrens.org
- Recruiting
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Jennifer Hawkins, MS, 513-636-7818, jennifer.hawkins@cchmc.org
Erin Chapman, BAS, 513-803-7482, erin.chapman@cchmc.org
- Recruiting
Texas Children's Hospital (Baylor College of Medicine)
Houston, Texas, 77030
Laurel Cavallo, 832-822-1053, children.network@bcm.edu
Cynthia Tsai, 832-822-3634, children.network@bcm.edu
- Completed
Riley Children's Hospital
Indianapolis, Indiana, 46202
- Recruiting
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Sirikorn Boonsawat, 323-361-2181, sboonsawat@chla.usc.edu
Christopher Gayer, MD
- Completed
Mount Sinai Medical Center
New York, New York, 10029
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Caitlin Griffiths, MS, 267-577-9888, griffithc2@chop.edu
Jordan Moreno, 267-425-1614, morenoj@chop.edu
- Completed
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
- Recruiting
University of Utah
Salt Lake City, Utah, 84113
Ann Rutherford, 801-585-9495, ann.rutherford@hsc.utah.edu
Natalie Fillerup, 801-587-5670, natalie.fillerup@hsc.utah.edu
- Completed
University of California at San Francisco
San Francisco, California, 94143
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Melissa Young, 206-987-1037, melissa.young@seattlechildrens.org
Teresa Chen, 206-987-6649, teresa.chen@seattlechildrens.org
- Completed
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: Biliary Atresia Study in Infants and Children (BASIC)
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 characterize the natural history of the older, non-transplanted child with BA.
Read the 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 2a: 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 2b: Health related quality of life will be impaired compared to healthy age matched children and relate to severity of illness.
Hypothesis 2c: 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.
- Conditions
- Biliary Atresia
- Keywords
- Biliary Atresia, Cholestasis
- Medical subject headings
- Biliary Atresia, Cholestasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Biliary atresia subjects who have their native liver |
What is being measured
The primary outcome is the main question the study is designed to answer.
To identify the gene or genes implicated in the etiology of BA
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.
Measured: Specimens for this aim are collected once during study, usually at baseline.
Secondary outcomes (1)
Define the natural history of the older, non-transplanted child with biliary atresia
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.
Measured: Observational information collected at entrance into study as well as at each yearly follow-up visit.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jun 2006 (estimated)
- Study start16 May 2006
- Primary completion31 May 2029 (estimated)
- Study completion31 May 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Biliary AtresiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 1,265 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arbor Research Collaborative for Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sanjiv Harpavat, MD, Study chair, Texas Children's/Baylor College of MedicineEd Doo, MD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)John C Magee, MD, Principal investigator, University of MichiganLisa Henn, PhD, Principal investigator, Arbor Research Collaborative for Health - Data Coordinating CenterKatrina Loh, MD, Study director, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00345553
- Sponsor's protocol ID
- BASIC Study - ChiLDReN Network
- Other IDs
- U01DK103149, U01DK103140, U01DK103135, U01DK084575, U01DK084538, U01DK084536, U01DK062503, U01DK062500
- Acronym
- BASIC
- Status last verified
- Jun 2026
- First submitted
- 27 Jun 2006
- Last updated
- 4 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The data will be transferred to NIDDK at the end of the study.
Publications
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.
PubMed 25015575
Study at a glance
From the registry record, last updated by the sponsor on 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Biliary Atresia Study in Infants and Children (BASIC)
- ClinicalTrials.gov ID
- NCT00345553
- Acronym / sponsor ID
- BASIC / BASIC Study - ChiLDReN Network
- Conditions
- Biliary Atresia
- Keywords
- Biliary Atresia, Cholestasis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 1,265 participants (estimated)
- Lead sponsor
- Arbor Research Collaborative for HealthUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- 16 May 2006 (actual)
- Primary completion
- 31 May 2029 (estimated)
- Study completion
- 31 May 2029 (estimated)
- First posted
- 28 Jun 2006 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 characterize the natural history of the older, non-transplanted child with BA.
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 2a: 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 2b: Health related quality of life will be impaired compared to healthy age matched children and relate to severity of illness.
Hypothesis 2c: 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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | –Biliary atresia subjects who have their native liver |
Who may qualify
- Ages
- 6 months to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (4 listed)
- 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 with their native liver.
- 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.
Key exclusion criteria (4 listed)
- 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.
- History of liver transplantation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
1 secondary outcome
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 2026.
- Derived from this study: PubMed 25015575Ng 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.
- Childhood Liver Disease Research Network (ChiLDReN) websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. The data will be transferred to NIDDK at the end of the study.
Related trials for Biliary Atresia
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingPhase 3
An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
Biliary Atresia
Active, not recruiting
Molecular Characterization for Understanding Biliary Atresia
Biliary Atresia
Active, not recruitingPhase 3
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)
RecruitingPhase 1
Umbilical Cord Mesenchymal Stem Cell Therapy for Biliary Atresia
Biliary Atresia, Kasai Operation
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sanjiv Harpavat, MD study chair
Texas Children's/Baylor College of Medicine
Ed Doo, MD study director
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
John C Magee, MD principal investigator
University of Michigan
16 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Biliary Atresia, run by Arbor Research Collaborative for Health at 16 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Melissa Sexton, BBA, CCRP (734-693-3811, melissa.sexton@arborresearch.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00345553, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Jun 2026.