NCT05932914Not yet recruiting
Liver Biopsy Following Gene Therapy For Hemophilia
Source: ClinicalTrials.gov · Last updated 22 Oct 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Study type
- Observational
- Enrollment
- 8 (estimated)
- First submitted
- 27 Jun 2023
- Start date
- Nov 2026
- Primary completion date
- Jan 2030
- Lead sponsor
- St. Jude Children's Research Hospital
- Diseases
- Haemophilia
- NCT number
- NCT05932914
What you commit to
- Study type: Observational
- Planned participants: 8
- From Nov 2026 to the primary completion date, Jan 2030
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Presence of FVIII inhibitor or FIX inhibitor (historical result can be used if done within 14 weeks of this liver biopsy)
Other illness
- 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
Lab values
- Platelet count \<140x10\^9/L
- INR \>1.5
- Abnormal kidney function with estimated GFR \<50 mL/min (calculated using the CKD-EPI equation)
Who the study is looking for
- 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
Age in the registry: 18 years to 80 years · Sex: Male · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| St. Jude Children's Research Hospital Coordinator of this site? Confirm its status | Memphis | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.