Status:
RECRUITING
Liver Biopsy In Haemophilia Gene Therapy
Lead Sponsor:
University College, London
Conditions:
Hemophilia B, Severe
Hemophilia A, Severe
Eligibility:
MALE
18-80 years
Phase:
NA
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 f...
Detailed Description
To better understand the consequences of AAV gene transfer patients will be recruited to undergo a liver biopsy. Patients will have endogenous FVIII:C/FIX:C expression at \>1% any time after gene tran...
Eligibility Criteria
Inclusion
- Inclusion criteria:
- Male and aged 18 to 80 years old
- 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)
- 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:
- Patients with a platelet count measured at \<140 x109/L
- 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.
- Patients with abnormal kidney function (estimated GFR \<50ml/min)
- Patients with a known allergy to iodine-based intravenous contrast agents
- Patients with a known allergy to local or general anaesthetic
- Patients with a known reaction to FVIII/FIX concentrate infusions
- Presence of FVIII or FIX inhibitor (done within 14 weeks of biopsy)
- Evidence of any bleeding disorder not related to haemophilia A or B
- Patients unable and unwilling to provide and sign an informed consent.
Exclusion
Key Trial Info
Start Date :
August 5 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 1 2025
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04817462
Start Date
August 5 2022
End Date
July 1 2025
Last Update
December 20 2024
Active Locations (1)
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1
Royal Free Hospital
London, United Kingdom, NW3 2QG