drugset / Trial / NCT03734588
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
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
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | SPK-8016 | Gene therapy (AAV / viral vector) | — | Intravenous |