Liver Biopsy Following Gene Therapy For Hemophilia
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexMale
Age18-80
SponsorSt. Jude Children's Research Hospital
About this trial
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.
Eligibility criteria
Qualifiers
Age ≥18 to 80 years
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
Disqualifiers
Any condition that, in the opinion of the investigator or sponsor of the ongoing clinical trial in which the patient is participating in, would prevent the patient from fully complying with the requirements of the clinical trial and/or would influence or interfere with evaluation and interpretation of subject safety or efficacy result of that ongoing clinical trial
Platelet count <140x10^9/L
INR >1.5
Abnormal kidney function with estimated GFR <50 mL/min (calculated using the CKD-EPI equation)
Trial design
Treatments tested in this trial
- Liver Biopsy
Treatment groups
8 Participants
are divided into 1 treatment groupSponsors and collaborators
St. Jude Children's Research Hospital
Lead sponsor
Methodist University Hospital
Collaborator
Source ClinicalTrials.gov