3 completed, 2 active not recruiting, 2 not yet recruiting, 1 enrolling by invitation. 5 Phase 2, 5 Phase 3, 4 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 3, Not yet recruiting 2 |
| Phase 3 | 5 | Active not recruiting 2, Completed 2, Enrolling by invitation 1 |
| Phase 1 | 4 | Not yet recruiting 2, Completed 1, Recruiting 1 |
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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 |
|---|---|---|---|---|---|---|
| A Safety and Efficacy Study Evaluating CTX112 in Subjects With Refractory Neurologic Autoimmune Disease CTX112 - Progressive Multiple Sclerosis (PMS), Neuromyelitis Optica | Phase 1 / Phase 2 | Not yet recruiting | CRISPR Therapeutics | 220 | 2026-11-01 | 2026-10-22 |
| A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD) CTX460 - Alpha-1 Antitrypsin Deficiency (AATD) | Phase 1 / Phase 2 | Not yet recruiting | CRISPR Therapeutics | 68 | 2026-10-31 | 2026-09-30 |
| A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease CTX112 - SLE (Systemic Lupus), Lupus Erythematosus, Systemic | Phase 1 | Recruiting | CRISPR Therapeutics | 80 | 2025-03-10 | 2026-10-26 |
| A Safety and Efficacy Study Evaluating CTX131 in Adult Subjects With Relapsed/Refractory Hematologic Malignancies CTX131 - T Cell Lymphoma, B Cell Lymphoma | Phase 1 / Phase 2 | Completed | CRISPR Therapeutics | 12 | 2024-08-13 | 2026-04-03 |
| 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 (5) · Hemoglobinopathies (5) · Anemia, Sickle Cell (3) · Genetic Diseases, Inborn (3) · Thalassemia (3) · beta-Thalassemia (3) · Autoimmune Diseases of the Nervous System (1) · Leukemia (1) · Leukemia, Myeloid, Acute (1) · Lupus Erythematosus, Systemic (1) · Lupus Nephritis (1) · Lymphoma, B-Cell (1) · Lymphoma, T-Cell (1) · Multiple Sclerosis, Chronic Progressive (1) · Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (1)
United States (7) · Germany (6) · Italy (5) · United Kingdom (5) · Canada (4) · Belgium (2) · Australia (1) · France (1) · Spain (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