Liver Biopsy Following Gene Therapy For Hemophilia
NCT05932914 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 8
Last updated 2026-05-20
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
- Hemophilia A
- Hemophilia B
Interventions
- PROCEDURE
-
Liver Biopsy
Standard transjugular liver biopsy under moderate sedation
Sponsors & Collaborators
-
Methodist University Hospital
collaborator UNKNOWN -
St. Jude Children's Research Hospital
lead OTHER
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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