Status: Recruiting
Liver Biopsy In Haemophilia Gene Therapy
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.
Participants needed: 10
Trial details
Age: 18-80Biological sex: MaleType: InterventionalSponsor: University College, LondonUpdated: Dec 20, 2024Locations: 1
Eligibility criteria
Male and aged 18 to 80 years old [+4]
Patients with a platelet count measured at <140 x109/L [+8]