1 active not recruiting, 1 completed, 1 recruiting, 1 suspended. 5 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Active not recruiting 1, Completed 1, Recruiting 1, Suspended 1, Unknown 1 |
| Phase 1 | 1 | Completed 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 |
|---|---|---|---|---|---|---|
| Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency RP-L301 - Pyruvate Kinase Deficiency | Phase 2 | Suspended | Rocket Pharmaceuticals Inc. | 10 | 2026-04-01 | 2026-04-16 |
| A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease RP-A501 - Danon Disease | Phase 2 | Recruiting | Rocket Pharmaceuticals Inc. | 18 | 2023-09-05 | 2026-10-06 |
| Gene Therapy for Fanconi Anemia, Complementation Group A RP-L102 - Fanconi Anemia Complementation Group A | Phase 2 | Active not recruiting | Rocket Pharmaceuticals Inc. | 5 | 2020-07-15 | 2025-12-22 |
| Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A RP-L102 - Fanconi Anemia Complementation Group A | Phase 2 | Unknown | Rocket Pharmaceuticals Inc. | 7 | 2019-11-28 | 2024-05-22 |
| A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I RP-L201 - Leukocyte Adhesion Defect - Type I | Phase 1 / Phase 2 | Completed | Rocket Pharmaceuticals Inc. | 9 | 2019-08-30 | 2023-11-15 |
Anemia (2) · Bone Marrow Failure Disorders (2) · Fanconi Syndrome (2) · Cardiomyopathies (1) · Cardiomyopathy, Hypertrophic (1) · Glycogen Storage Disease Type IIb (1) · Hypertrophy, Left Ventricular (1) · Leukocyte adhesion deficiency type 1 (1) · Lysosomal Storage Diseases (1) · Pyruvate Kinase Deficiency of Red Cells (1)
Methods (1) · Therapeutics (1)
United States (4) · Spain (3) · United Kingdom (3) · Germany (1) · Italy (1)
Ami Shah, MD (1) · Barry Greenberg, MD (1) · Claire Booth, MBBS, PhD, MSc (1) · Donald B Kohn, MD (1) · Joseph Rossano, MD (1) · Julian Sevilla Navarro, MD, PhD (1) · Margaret MacMillan, MD, MSc (1) · Maria Chitty-Lopez, MD (1) · Rajni Agarwal, MD (1)
2018 (1)