| A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy (NCT07137338) | Rocket Pharmaceuticals Inc. | 2026-06-01 |
| BAG3-DCM Natural History Study (NCT07486752) | Rocket Pharmaceuticals Inc. | 2026-05-01 |
| Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency (NCT06422351) | Rocket Pharmaceuticals Inc. | 2026-04-01 |
| PKP2-ACM Natural History Study (NCT06644742) | Rocket Pharmaceuticals Inc. | 2026-03-01 |
| Danon Disease Natural History Study (NCT06214507) | Rocket Pharmaceuticals Inc. | 2023-12-20 |
| A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease (NCT06092034) | Rocket Pharmaceuticals Inc. | 2023-09-05 |
| A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) (NCT05885412) | Rocket Pharmaceuticals Inc. | 2023-08-29 |
| Natural History of Danon Disease (NCT05548855) | Rocket Pharmaceuticals Inc. | 2022-09-26 |
| Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) (NCT06282432) | Rocket Pharmaceuticals Inc. | 2022-03-09 |
| Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 (NCT07527975) | Rocket Pharmaceuticals Inc. | 2022-02-01 |
| A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis (NCT04525352) | Rocket Pharmaceuticals Inc. | 2020-11-19 |
| Gene Therapy for Fanconi Anemia, Complementation Group A (NCT04248439) | Rocket Pharmaceuticals Inc. | 2020-07-15 |
| Gene Therapy for Pyruvate Kinase Deficiency (PKD) (NCT04105166) | Rocket Pharmaceuticals Inc. | 2020-07-06 |
| Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A (NCT04069533) | Rocket Pharmaceuticals Inc. | 2019-11-28 |
| A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I (NCT03812263) | Rocket Pharmaceuticals Inc. | 2019-08-30 |
| Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B (NCT03882437) | Rocket Pharmaceuticals Inc. | 2019-04-17 |
| A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I (NCT03825783) | Rocket Pharmaceuticals Inc. | 2019-04-15 |
| A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A (NCT03814408) | Rocket Pharmaceuticals Inc. | 2019-01-11 |