Gene Therapy for Chinese Hemophilia A
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Gene Therapy
- Hemophilia A
- Type
- Interventional
- Phase
- Not Applicable
- Design
- Allocation: N/AIntervention Model: Single Group AssignmentMasking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 18 years and 125 years
- Gender
- Only males
Description
GS001 is a open- label, non- randomized, uncontrolled, study to evaluate the safety, tolerability and kinetics of GS001 in hemophilia A subjects with residual FVIII levels<1IU/dl. Three patients will be enrolled sequentially every 3 weeks or more between cohorts and administered with single infusion...
GS001 is a open- label, non- randomized, uncontrolled, study to evaluate the safety, tolerability and kinetics of GS001 in hemophilia A subjects with residual FVIII levels<1IU/dl. Three patients will be enrolled sequentially every 3 weeks or more between cohorts and administered with single infusion of GS001.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 dosing to the second subject will not be performed until acquiring the approve from independent safety committee.The dose levels are as follows: 2×10^12 vg/kg 6×10^12 vg/kg 2. 2×10^13 vg/kg Subjects will provide informed consent and then undergo screening assessments up to 4-8weeks prior administration of GS001. All subjects will undergo 52(+- 2) weeks safety observation.
Tracking Information
- NCT #
- NCT04728841
- Collaborators
- Not Provided
- Investigators
- Principal Investigator: Lei Zhang, MD Chinese Academy of Medical Science and Blood Disease Hospital
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