Liver Biopsy In Haemophilia Gene Therapy
Recruiting
N/A
Interventional Study
Hemophilia B, Severe
Hemophilia A, Severe
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 18 – 80
- Sex
- Male
- Study type
- Interventional
- Purpose
- Basic Science
- Participants needed
- 10 (estimated)
- Sponsor
- University College, London · Other
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About This Trial
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 episoma…
Trial Locations
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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 …
Contacts