Clinical trial
A Safety and Efficacy Study Evaluating CTX001 in Participants With…
NCT03655678 · Vertex Pharmaceuticals Incorporated, Industry · Completed
Key facts
| NCT number | NCT03655678 |
|---|---|
| Title | A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia |
| Official 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, Industry |
| Collaborators | CRISPR Therapeutics Industry |
| Status | Completed |
| Phase | Phase 2/Phase 3 |
| Study type | Interventional |
| Conditions | Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies |
| Intervention type | Biological |
| Interventions | CTX001 |
| Enrollment | 59 |
| Start date | Sep 14, 2018 |
| Primary completion | Nov 13, 2025 |
| Completion | Nov 13, 2025 |
| First posted | Aug 31, 2018 |
| Last update posted | Dec 17, 2025 |
| Countries | Canada, Germany, Italy, United Kingdom, United States |
At a glance
- 10–99 Study sites
- No Results posted
- None Linked FDA submissions
Counts are shown as ranges. Exact figures, trends and every underlying record are available in Orca1.
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Source: U.S. National Library of Medicine (NLM), ClinicalTrials.gov · Source date: