Liver Biopsy Following Gene Therapy For Hemophilia
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.
Description
This is a prospective cohort study evaluating the effect of AAV-mediated gene therapy on the liver in adult patients with hemophilia A or hemophilia B who have previously been treated with a FVIII/FIX gene-containing AAV-vector for liver-targeted gene transfer. To better understand the effect of AAV-mediated gene transfer on the liver, eligible participants can allow the use and analysis of already existing liver tissue samples, taken and preserved after having received gene therapy. Alternatively, eligible participants can consent to providing a new liver tissue sample which will be obtained…
Eligibility
- Age range
- 18–80 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: * Age ≥18 to 80 years * Patients, who were enrolled and treated in one of the following clinical trials: * AGT4HB (EudraCT number: 2005-005711-17; NCT00979238) - FIX AAV gene therapy trial (sponsor: St. Jude Children's Research Hospital) * GO8 (EudraCT number:2014-003880-38; NCT02576795) - FVIII AAV gene therapy trial (sponsor: University College, London) * Able to give informed consent * Able to comply with study requirements Exclusion Criteria (Do not apply to participants who will not undergo liver biopsy, and have leftover liver tissue from a previous biopsy proc…
Interventions
- ProcedureLiver Biopsy
Standard transjugular liver biopsy under moderate sedation
Location
- St. Jude Children's Research HospitalMemphis, Tennessee