NCT04817462 Liver Biopsy In Haemophilia Gene Therapy
| NCT ID | NCT04817462 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University College, London |
| Condition | Hemophilia B, Severe |
| Study Type | INTERVENTIONAL |
| Enrollment | 10 participants |
| Start Date | 2022-08-05 |
| Primary Completion | 2025-07-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
This trial targets 10 participants in total. It began in 2022-08-05 with a primary completion date of 2025-07-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
To perform a liver biopsy in haemophilia A and B patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer following AAV mediated gene transfer. This is to obtain tissue for analysis, to understand if FIX/FVIII transgenic protein expression is mediated by AAV proviral DNA that is integrated into the host cell DNA or if stable expression in humans is mediated by episomal maintained AAV genome.
Eligibility Criteria
Inclusion criteria: 1. Male and aged 18 to 80 years old 2. Patients who were enrolled and treated in one of the following clinical trials at Royal Free Hospital: * AGT4HB (EudraCT No 2005-005711-17) - FIX AAV gene therapy trial (Sponsor: St Jude Children's Research Hospital) * GO-8 (EudraCT No 2016-000925-38) - FVIII AAV gene therapy trial (Sponsor: UCL) * FLT180a-01 (EudraCT: 2017-000852-24) - FIX AAV gene therapy trial ((Sponsor: UCL) \[now enrolled in long term follow up study FLT180a-04 (EudraCT No 2017-005080-40) (Sponsor: Freeline Therapeutics Ltd) 3. Patients with endogenous FVIII:C/FIX:C expression at \>1% any time after gene transfer, associated with normal prothrombin (PT) and thrombin times (TT) as determined in a coagulation assay. Exclusion Criteria: 1. Patients with a platelet count measured at \<140 x109/L 2. Any condition that, in the opinion of the investigator or Sponsor would prevent the patient from fully complying with the requirements of the study and/or would influence or interfere with evaluation and interpretation of subject safety or efficacy result. 3. Patients with abnormal kidney function (estimated GFR \<50ml/min) 4. Patients with a known allergy to iodine-based intravenous contrast agents 5. Patients with a known allergy to local or general anaesthetic 6. Patients with a known reaction to FVIII/FIX concentrate infusions 7. Presence of FVIII or FIX inhibitor (done within 14 weeks of biopsy) 8. Evidence of any bleeding disorder not related to haemophilia A or B 9. Patients unable and unwilling to provide and sign an informed consent.
Contact & Investigator
Paul Batty, MBBS MRCP
PRINCIPAL INVESTIGATOR
Royal Free Hospital NHS Foundation Trust
Frequently Asked Questions
Who can join the NCT04817462 clinical trial?
This trial is open to male participants only, aged 18 Years or older, up to 80 Years, studying Hemophilia B, Severe. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT04817462 currently recruiting?
Yes, NCT04817462 is actively recruiting participants. Contact the research team at paul.batty@ucl.ac.uk for enrollment information.
Where is the NCT04817462 trial being conducted?
This trial is being conducted at London, United Kingdom.
Who is sponsoring the NCT04817462 clinical trial?
NCT04817462 is sponsored by University College, London. The principal investigator is Paul Batty, MBBS MRCP at Royal Free Hospital NHS Foundation Trust. The trial plans to enroll 10 participants.