Studies liver tissue in adults with hemophilia after gene therapy
Examines liver biopsy samples from adults with hemophilia previously treated with gene therapy.
Plain-language title generated from the registry record
Registry title: Liver Biopsy Following Gene Therapy For Hemophilia
Key facts
- Where
- Memphis, United StatesSee the site
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 80 years old?
Source: NCT05932914, who can take part
Where in United States
- St. Jude Children's Research HospitalMemphis, United StatesNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- St. Jude Children's Research Hospital
- Trial code
- NCT05932914
- Record updated
- 22 October 2026
- Original description
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.Read full original descriptionShow less
Liver Biopsy Following Gene Therapy For Hemophilia
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.
Inclusion: 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: Presence of FVIII inhibitor or FIX inhibitor (historical result can be used if done within 14 weeks of this liver biopsy); 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; Known allergy to iodine-based intravenous contrast agents; Known allergy to local or general anesthetics; Known allergic reaction to FVIII/FIX concentrate infusions; Evidence of any bleeding disorder other than hemophilia A or B; Platelet count <140x10\^9/L; INR >1.5; Abnormal kidney function with estimated GFR <50 mL/min (calculated using the CKD-EPI equation).