Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Hemophilia A
- Type
- Interventional
- Phase
- Phase 1
- Design
- Allocation: N/AIntervention Model: Single Group AssignmentIntervention Model Description: Open-label, nonrandomized, single-center phase I cohort studyMasking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Between 18 years and 125 years
- Gender
- Only males
Description
This is an open label, nonrandomized, single center, phase I cohort study, involving reduced intensity conditioning, followed by a single infusion of autologous CD34+PBSC, transduced with a lentiviral vector (-889ITGA2B-BDDFVIII-WPTS (MUT6)(VSVg)) also known as (Pleightlet(MUT6)) encoding the B doma...
This is an open label, nonrandomized, single center, phase I cohort study, involving reduced intensity conditioning, followed by a single infusion of autologous CD34+PBSC, transduced with a lentiviral vector (-889ITGA2B-BDDFVIII-WPTS (MUT6)(VSVg)) also known as (Pleightlet(MUT6)) encoding the B domain deleted from of human coagulation factor VIII (BDDFVIII) in up to five hemophilia A patients with inhibitors to FVIII.
Tracking Information
- NCT #
- NCT03818763
- Collaborators
- Not Provided
- Investigators
- Principal Investigator: Parmeswaran Hari, MD Froedtert Hosptial and Medical College of Wisconsin
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