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RECRUITING
NCT04817462
NA

Liver Biopsy In Haemophilia Gene Therapy

Sponsor: University College, London

View on ClinicalTrials.gov

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

Interventions

PROCEDURE

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