drugset / Trial / NCT03001830
Gene Therapy for Haemophilia A.
Phase 1/2
Active not recruiting
14 enrolled
University College, London
Medical Research Council · collab
NaSingle-groupOpen-labelTreatment
Summary
The GO-8 study focuses on assessing safety and efficacy of gene therapy for patients with severe haemophilia A
Timeline
- Start
- 2017-06-14
- Primary completion
- 2029-01
- Completion
- 2029-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV2/8-HLP-FVIII-V3 | Gene therapy (AAV / viral vector) | 6e+11 vg/kg | Other |
| Subject | AAV2/8-HLP-FVIII-V3 | Gene therapy (AAV / viral vector) | 2e+12 vg/kg | Other |
| Subject | AAV2/8-HLP-FVIII-V3 | Gene therapy (AAV / viral vector) | 6e+12 vg/kg | Other |