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Liver Biopsy In Haemophilia Gene Therapy
Sponsor: University College, London
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.
Key Details
Gender
MALE
Age Range
18 Years - 80 Years
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2022-08-05
Completion Date
2025-07-01
Last Updated
2024-12-20
Healthy Volunteers
No
Conditions
Interventions
Liver biopsy
The study population is patients with either haemophilia A or B who have previously been administered gene therapy treatment in one of three specific gene therapy clinical trials. In this study they will have a liver biopsy performed to take up to 3 samples for laboratory analysis.
Locations (1)
Royal Free Hospital
London, United Kingdom