Clinical trial
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
NCT05477563 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 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Phase
- Phase 3
- Overall status
- Recruiting
- Start date
- August 2, 2022
- Completion date
- June 9, 2027
- Enrollment
- 26 (estimated)
- Sites
- 6
Conditions
As the registry lists them; a condition with its own page is linked.
beta-thalassemia, genetic diseases, inborn, hematologic diseases, hemoglobinopathies, sickle cell anemia, sickle cell disease, thalassemia