Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Sickle Cell Disease
- Type
- Interventional
- Phase
- Phase 1Phase 2
- Design
- Allocation: N/AIntervention Model: Single Group AssignmentIntervention Model Description: A open label, non-randomized, first-in-patient, phase I/II, proof-of-concept study following subjects for two years after transplantation of either genome-edited autologous HSPC investigational drug product. The study consist of 3 parts - Part A include treatment of adults with OTQ923; Part B include treatment of adults with HIX763; Part C include treatment of kids 2-17 years old with either OTQ923 or HIX763Masking: None (Open Label)Masking Description: The is an open-label study.Primary Purpose: Treatment
Participation Requirements
- Age
- Between 2 years and 40 years
- Gender
- Both males and females
Description
Not Provided
Not Provided
Tracking Information
- NCT #
- NCT04443907
- Collaborators
- Not Provided
- Investigators
- Not Provided
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