Liver Biopsy Following Gene Therapy For Hemophilia

NCT05932914 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 8

Last updated 2026-05-20

No results posted yet for this study

Summary

This observational study will obtain liver biopsy samples and evaluate the long-term effect of adeno-associated virus (AAV)-mediated gene therapy on the liver tissue in adult patients with hemophilia A or hemophilia B who have previously been treated with a factor VIII or factor IX gene-containing AAV-vector for liver-targeted gene transfer. Participants are from a cohort of patients treated with AAV-mediated gene transfer and at least 6 months after vector infusion.

Conditions

Interventions

PROCEDURE

Liver Biopsy

Standard transjugular liver biopsy under moderate sedation

Sponsors & Collaborators

Principal Investigators

  • Ulrike Reiss, MD · St. Jude Children's Research Hospital

Eligibility

Min Age
18 Years
Max Age
80 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-30
Primary Completion
2030-01-31
Completion
2030-01-31

Countries

  • United States

Study Locations

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Entities

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05932914 on ClinicalTrials.gov