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Liver Biopsy Following Gene Therapy For Hemophilia
Sponsor: St. Jude Children's Research Hospital
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.
Key Details
Gender
MALE
Age Range
18 Years - 80 Years
Study Type
OBSERVATIONAL
Enrollment
8
Start Date
2026-05
Completion Date
2030-01
Last Updated
2026-04-08
Healthy Volunteers
No
Conditions
Interventions
Liver Biopsy
Standard transjugular liver biopsy under moderate sedation
Locations (1)
St. Jude Children's Research Hospital
Memphis, Tennessee, United States