Actively Recruiting

All Genders
ID03273049

Coordinating Center - Mapping Disease Pathways for Biliary Atresia

Led by University of Pittsburgh · Updated on 2025-09-09

1100

Participants Needed

1

Research Sites

239 weeks

Total Duration

On this page

Sponsors

U

University of Pittsburgh

Lead Sponsor

N

National Institutes of Health (NIH)

Collaborating Sponsor

AI-Summary

What this Trial Is About

Biliary atresia is a condition where bile ducts outside the liver fail to develop properly, leading to liver failure in newborns. This condition is a leading cause of liver transplants in children worldwide. The study aims to understand the genetic and developmental causes of biliary atresia by analyzing DNA samples from affected children and their family members to identify key genes and pathways involved. The study will collect and analyze genetic information from around 1100 children diagnosed with biliary atresia and their biological parents or siblings, if available. Samples will be studied using human liver tissue and animal models to validate findings. The research involves collaboration among major pediatric liver transplant centers across different countries and uses advanced bioinformatics tools to explore disease pathways. Participants will provide DNA samples and medical data during the study. Researchers will monitor genetic pathways related to biliary atresia over a period of up to two years, with some outcomes assessed over four years or more. The study focuses on understanding disease mechanisms to help develop better treatments and management strategies for this rare childhood liver disease. Participation does not involve drug treatments or interventions but contributes to important genetic research.

CONDITIONS

Brief Title

Mapping Disease Pathways for Biliary Atresia

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Living individuals diagnosed with biliary atresia
  • Have received or are about to receive a liver transplant
  • Enrolled from participating centers including Children's Hospital of Pittsburgh, Kings College Hospital, Children's Hospital of Birmingham, and Hospital S�edrio-Liban�es
Not Eligible

You will not qualify if you...

  • Children in the care of the state are not eligible
  • Patients under temporary or informal guardianship cannot participate

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Sample Collection

Duration - Up to 2 years

Participants provide biological samples including DNA and liver tissue for analysis of genetic susceptibility and disease pathways related to biliary atresia.

1 to 2 visits depending on sample availability

Long-term Monitoring

Duration - Upwards of 4 years

Participants are observed over several years to study disease progression and genetic predisposition to biliary atresia.

Periodic visits for follow-up assessments

Trial Site Locations

Total: 1 location

1

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States, 15224

Actively Recruiting

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Research Team

M

Morgan L Paul, BSN

D

Daniel Pieratt, MPA

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

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