Liver Biopsy Following Gene Therapy For Hemophilia

Launched by ST. JUDE CHILDREN'S RESEARCH HOSPITAL · Jun 27, 2023

Keywords

ClinConnect Summary

This clinical trial is studying the long-term effects of a specific type of gene therapy on the liver in adults with hemophilia A or hemophilia B. The researchers will collect liver tissue samples from participants who have already received gene therapy using a special virus to help their bodies produce the clotting factors they need. The goal is to understand how this gene therapy impacts liver health over time, particularly in patients who have shown stable improvement in their condition for at least six months after treatment.

To be eligible for this study, participants need to be between 18 and 80 years old and must have previously participated in specific clinical trials related to their hemophilia treatment. They should also have a certain level of clotting factors in their blood after receiving the gene therapy. Participants can expect to have a liver biopsy, which is a procedure to take a small sample of liver tissue for testing. It's important to note that people with specific health conditions, such as low platelet counts or certain allergies, may not be able to join the study. The trial is not yet recruiting participants, so those interested will need to wait for further announcements.

Gender

MALE

Eligibility criteria

• 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:2016-000925; NCT03001830) - FVIII AAV gene therapy trial (sponsor: University College, London)
• • Patients with endogenous expression of FVIII/FIX at \>1% after gene transfer that is stably maintained for more than six months after vector infusion
• • Able to give informed consent
• • Able to comply with study requirements
• Exclusion Criteria:
• • 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 \<100x10\^9/L
• • INR \>1.5
• • Abnormal kidney function with estimated GFR \<50 mL/min (calculated using the CKD-EPI equation)
• • Known allergy to iodine-based intravenous contrast agents
• • Known allergy to local or general anesthetics
• • Known allergic reaction to FVIII/FIX concentrate infusions
• • Presence of FVIII/FIX inhibitor
• • Evidence of any bleeding disorder other than hemophilia A or B