Study Summary
IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with \<1 IU/dl residual FVIII levels.
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Injection of GS001GENETIC
Patients will be enrolled sequentially every 3 weeks or more between cohorts. Dose escalation may occur after a single patient has been safely dosed if the resulting FVIII activity at Week 3 is \< 5 IU/dL.The dose levels are as follows:
1. 2×10\^12 vg/kg
2. 6×10\^12vg/kg or other recommended doses
3. 2×10\^13 vg/kg or other recommended doses
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Chinese Academy of Medical Science and Blood Disease Hospital | Tianjin | Tianjin Municipality | China |