drugset / Trial / NCT03734588

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

NCT03734588 ↗

Phase 1/2 Completed 4 enrolled Spark Therapeutics, Inc.
NaSingle-groupOpen-labelTreatment

Summary

SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII.

Timeline

Start
2019-01-30
Primary completion
2020-10-14
Completion
2023-01-19

Drugs

EvaluationDrugModalityDoseRoute
Subject SPK-8016 Gene therapy (AAV / viral vector) — Intravenous

Indications