NCT03273049

Brief Summary

This project will primarily evaluate the developmental/genetic basis of biliary atresia, the most common cause of liver failure at birth, and which accounts of half of all liver transplants performed worldwide in children.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1,100

participants targeted

Target at P75+ for all trials

Timeline
112mo left

Started Jul 2016

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress52%
Jul 2016Jul 2035

Study Start

First participant enrolled

July 21, 2016

Completed
1.1 years until next milestone

First Submitted

Initial submission to the registry

September 1, 2017

Completed
5 days until next milestone

First Posted

Study publicly available on registry

September 6, 2017

Completed
13.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 21, 2030

Expected
4.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 21, 2035

Last Updated

September 9, 2025

Status Verified

September 1, 2025

Enrollment Period

14.4 years

First QC Date

September 1, 2017

Last Update Submit

September 5, 2025

Conditions

Biliary Atresia

Outcome Measures

Primary Outcomes (1)

  • Genomic pathways of BA

    Main project outcome will consist of pathways comprising multiple susceptibility genes involved in morphogenesis of the liver and other organs, which explain the complex phenotype of BA.

    up to two years

Secondary Outcomes (1)

  • Predisposition of BA

    upwards of four years to achieve this outcome measure

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Individuals who have had a liver transplantation due to a diagnosis of biliary atresia.

You may qualify if:

  • living individuals who were diagnosed with Biliary Atresia and received or are about to receive a liver transplant from multiple participating centers (Children's Hospital of Pittsburgh, Kings College Hospital, Children's Hospital of Birmingham, and Hospital Sírio-Libanês).

You may not qualify if:

  • No child participant in the care of the state will be enrolled, nor will patients in the care of temporary or informal guardians be enrolled

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Samples with DNA: blood, saliva or liver tissue.

MeSH Terms

Conditions

Biliary Atresia

Condition Hierarchy (Ancestors)

Bile Duct DiseasesBiliary Tract DiseasesDigestive System DiseasesDigestive System AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Morgan L Paul, BSN

CONTACT

4126928472Morgan.Paul2@upmc.edu

Daniel Pieratt, MPA

CONTACT

4126926692pierattdw@upmc.edu

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Rakesh Sindhi, MD, Professor of Surgery

Study Record Dates

First Submitted

September 1, 2017

First Posted

September 6, 2017

Study Start

July 21, 2016

Primary Completion (Estimated)

December 21, 2030

Study Completion (Estimated)

July 21, 2035

Last Updated

September 9, 2025

Record last verified: 2025-09

Locations

US(1)