2 active not recruiting, 2 completed, 2 recruiting, 1 enrolling by invitation. 7 Phase 3, 3 Phase 2, 2 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 3 | 7 | Active not recruiting 2, Completed 2, Enrolling by invitation 1, Recruiting 1, Withdrawn 1 |
| Phase 2 | 3 | Completed 2, Recruiting 1 |
| Phase 1 | 2 | Not yet recruiting 1, Recruiting 1 |
Follow CTX001: new trials by email Literature Review of CTX001 trials Research Report on CTX001
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Evaluation of Efficacy and Safety of a Single Dose of Exa-cel in Participants With Severe Sickle Cell Disease, βS/ βC Genotype Exa-cel - Sickle Cell Disease | Phase 3 | Withdrawn | Vertex Pharmaceuticals Incorporated | 2027-07-31 | 2026-03-25 | |
| Study of CTX001 in Adults With Anemia Due to Non-Dialysis-Dependent Chronic Kidney Disease Placebo, CTX001, Ferrous Sulfate - Chronic Kidney Disease, Anemia | Phase 1 | Not yet recruiting | Cajal Therapeutics Inc. | 24 | 2026-11-01 | 2026-10-28 |
| An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA) CTx001 - Geographic Atrophy Secondary to Age-related Macular Degeneration | Phase 1 / Phase 2 | Recruiting | Complement Therapeutics | 75 | 2025-12-30 | 2026-10-16 |
| Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease CTX001 - Beta-Thalassemia, Thalassemia | Phase 3 | Recruiting | Vertex Pharmaceuticals Incorporated | 26 | 2022-08-02 | 2026-03-23 |
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) CTX001 - Beta-Thalassemia, Thalassemia | Phase 3 | Active not recruiting | Vertex Pharmaceuticals Incorporated | 16 | 2022-05-03 | 2026-07-01 |
| Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) CTX001 - Sickle Cell Disease, Hydroxyurea Failure | Phase 3 | Active not recruiting | Vertex Pharmaceuticals Incorporated | 13 | 2022-05-02 | 2026-07-01 |
| A Long-term Follow-up Study in Participants Who Received CTX001 CTX001 - Beta-Thalassemia, Thalassemia | Phase 3 | Enrolling by invitation | Vertex Pharmaceuticals Incorporated | 160 | 2021-01-20 | 2026-08-11 |
| A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease Exa-cel - Sickle Cell Disease, Hematological Diseases | Phase 2 / Phase 3 | Completed | Vertex Pharmaceuticals Incorporated | 63 | 2018-11-27 | 2026-09-16 |
| A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia CTX001 - Beta-Thalassemia, Thalassemia | Phase 2 / Phase 3 | Completed | Vertex Pharmaceuticals Incorporated | 59 | 2018-09-14 | 2025-12-17 |
Hematologic Diseases (6) · Hemoglobinopathies (6) · Anemia, Sickle Cell (5) · Genetic Diseases, Inborn (4) · Thalassemia (4) · beta-Thalassemia (4) · Anemia (1) · Renal Insufficiency, Chronic (1)
Vertex Pharmaceuticals Incorporated (7) · Cajal Therapeutics Inc. (1) · Complement Therapeutics (1)
United States (7) · Germany (6) · Italy (6) · United Kingdom (6) · Canada (4) · Belgium (2) · Australia (1) · France (1) · Saudi Arabia (1)
PMID 33283989, PMID 34175041, PMID 40857358, PMID 40862696, PMID 42252696, PMID 42274009, PMID 32873924, PMID 36123234, PMID 36860093, PMID 36975201, PMID 38657265, PMID 38661449, PMID 38866240
2023 (1)