Clinical trial
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With…
NCT05329649 · Vertex Pharmaceuticals Incorporated, Industry · Recruiting
Key facts
| NCT number | NCT05329649 |
|---|---|
| Title | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) |
| Official title | A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease |
| Lead sponsor | Vertex Pharmaceuticals Incorporated, Industry |
| Collaborators | CRISPR Therapeutics Industry |
| Status | Recruiting |
| Phase | Phase 3 |
| Study type | Interventional |
| Conditions | Sickle Cell Disease, Hydroxyurea Failure, Hydroxyurea Intolerance, Hemoglobinopathies, Hematological Diseases |
| Intervention type | Biological |
| Interventions | CTX001 |
| Enrollment | 15 |
| Start date | May 2, 2022 |
| Primary completion | May 31, 2026 |
| Completion | May 31, 2026 |
| First posted | Apr 15, 2022 |
| Last update posted | Oct 15, 2025 |
| Countries | 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: