1 Study Matches
Dose-finding study of SPK-8016 gene therapy in patients with hemophilia A to support future evaluations in individuals with FVIII inhibitors.
SPK-8016-101 is a Phase 1/2a, open-label, non-randomized, dose-finding study designed to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with clinically severe hemophilia A and no measurable inhibitor against FVIII.
18 year(s) or older
Inclusion Criteria:Clinically severe Hemophilia A
Previous exposure to FVIII therapy
Have >150 exposure days to any recombinant and/or plasma-derived FVIII concentrates
No history of hypersensitivity or anaphylaxis with any FVIII or IV immunoglobulin product
No measurable inhibitor against FVIII
Exclusion Criteria:Active Hepatitis B or C
Current use of antiviral therapy to treat Hepatitis B or C
Documented significant liver disease
Serological evidence of HIV-1 or HIV-2
Anti-AAV-Spark neutralizing antibody titers >1:5