Clinical trial
A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia
NCT03655678 is registered in ClinicalTrials.gov with Vertex Pharmaceuticals Incorporated as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Phase
- Phase 2/3
- Overall status
- Completed
- Start date
- September 14, 2018
- Completion date
- November 13, 2025
- Enrollment
- 59 (actual)
- Sites
- 14
Conditions
As the registry lists them; a condition with its own page is linked.