Clinical trial
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle…
NCT03745287 · Vertex Pharmaceuticals Incorporated, Industry · Completed
Key facts
| NCT number | NCT03745287 |
|---|---|
| Title | A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease |
| Official title | A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease |
| Lead sponsor | Vertex Pharmaceuticals Incorporated, Industry |
| Collaborators | CRISPR Therapeutics Industry |
| Status | Completed |
| Phase | Phase 2/Phase 3 |
| Study type | Interventional |
| Conditions | Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies |
| Intervention type | Biological |
| Interventions | CTX001 |
| Enrollment | 63 |
| Start date | Nov 27, 2018 |
| Primary completion | Jul 7, 2025 |
| Completion | Jul 7, 2025 |
| First posted | Nov 19, 2018 |
| Last update posted | Aug 11, 2025 |
| Countries | Belgium, Canada, France, 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: