Clinical trial
A Long-term Follow-up Study in Participants Who Received CTX001
NCT04208529 · Vertex Pharmaceuticals Incorporated, Industry · Enrolling by invitation
Key facts
| NCT number | NCT04208529 |
|---|---|
| Title | A Long-term Follow-up Study in Participants Who Received CTX001 |
| Official title | A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001) |
| Lead sponsor | Vertex Pharmaceuticals Incorporated, Industry |
| Collaborators | CRISPR Therapeutics Industry |
| Status | Enrolling by invitation |
| Phase | Phase 3 |
| Study type | Interventional |
| Conditions | Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia |
| Intervention type | Biological |
| Interventions | CTX001 |
| Enrollment | 160 |
| Start date | Jan 20, 2021 |
| Primary completion | Sep 30, 2039 |
| Completion | Sep 30, 2039 |
| First posted | Dec 23, 2019 |
| Last update posted | Nov 3, 2025 |
| Countries | Belgium, 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: