5 recruiting, 4 completed, 3 active not recruiting, 3 unknown. 8 Phase 1, 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 8 | Completed 3, Recruiting 2, Terminated 1, Unknown 1, Withdrawn 1 |
| Phase 2 | 5 | Active not recruiting 1, Completed 1, Recruiting 1, Suspended 1, Unknown 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 |
|---|---|---|---|---|---|---|
| BAG3-DCM Natural History Study Dilated Cardiomyopathy (DCM), Cardiovascular Diseases | Not yet recruiting | Rocket Pharmaceuticals Inc. | 30 | 2026-12-01 | 2026-10-19 | |
| PKP2-ACM Natural History Study Cardiomyopathies, Heart Diseases | Recruiting | Rocket Pharmaceuticals Inc. | 36 | 2026-05-15 | 2026-10-14 | |
| A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3) - Dilated Cardiomyopathy (DCM) | Phase 1 | Recruiting | Rocket Pharmaceuticals Inc. | 8 | 2026-04-17 | 2026-10-01 |
| 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 |
| Danon Disease Natural History Study No intervention - Danon Disease | Recruiting | Rocket Pharmaceuticals Inc. | 60 | 2023-12-20 | 2026-10-19 | |
| 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 |
| A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) RP-A601 - PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) | Phase 1 | Recruiting | Rocket Pharmaceuticals Inc. | 9 | 2023-08-29 | 2026-04-16 |
| Natural History of Danon Disease Heart Transplant, Cardiac Assistive Device - Danon Disease | Completed | Rocket Pharmaceuticals Inc. | 59 | 2022-09-26 | 2023-11-21 | |
| Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) Leukocyte Adhesion Deficiency | Active not recruiting | Rocket Pharmaceuticals Inc. | 9 | 2022-03-09 | 2025-12-18 | |
| Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 RP-L102 - Fanconi Anemia | Enrolling by invitation | Rocket Pharmaceuticals Inc. | 14 | 2022-02-01 | 2026-04-14 | |
| A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis RP-L401 - Infantile Malignant Osteopetrosis | Phase 1 | Terminated | Rocket Pharmaceuticals Inc. | 1 | 2020-11-19 | 2022-07-13 |
| 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 |
| Gene Therapy for Pyruvate Kinase Deficiency (PKD) RP-L301 - Pyruvate Kinase Deficiency | Phase 1 | Completed | Rocket Pharmaceuticals Inc. | 4 | 2020-07-06 | 2025-09-05 |
| Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy Safety and efficacy assessments - Fanconi Anemia Complementation Group A, Fanconi Anemia | Active not recruiting | Rocket Pharmaceuticals Inc. | 9 | 2020-06-01 | 2026-04-13 | |
| 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 |
| Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B RP-A501 - Danon Disease | Phase 1 | Completed | Rocket Pharmaceuticals Inc. | 7 | 2019-04-17 | 2026-10-06 |
| A Gene Therapy 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 | Withdrawn | Rocket Pharmaceuticals Inc. | 2019-04-15 | 2021-12-07 | |
| A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A RP-L102 - Fanconi Anemia Complementation Group A | Phase 1 | Unknown | Rocket Pharmaceuticals Inc. | 2 | 2019-01-11 | 2020-11-24 |
| The Natural History of Danon Disease Danon Disease | Unknown | University of California, San Diego | 200 | 2018-11-30 | 2022-05-12 |
Cardiomyopathies (5) · Glycogen Storage Disease Type IIb (5) · Anemia (4) · Bone Marrow Failure Disorders (4) · Fanconi Syndrome (4) · Cardiovascular Diseases (3) · Genetic Diseases, Inborn (3) · Heart Diseases (3) · Leukocyte adhesion deficiency type 1 (3) · Arrhythmogenic Right Ventricular Dysplasia (2) · Cardiomyopathy, Dilated (2) · Cardiomyopathy, Hypertrophic (2) · Fanconi Anemia (2) · Pyruvate Kinase Deficiency of Red Cells (2) · Anemia, Hemolytic (1)
Heart Transplantation (1) · Methods (1) · Safety (1) · Therapeutics (1)
United States (15) · Spain (10) · United Kingdom (6) · Italy (3) · Germany (2) · Australia (1) · Czechia (1) · France (1) · Ireland (1) · Israel (1) · Netherlands (1)
Barry Greenberg, MD (3) · Ami Shah, MD (2) · Claire Booth, MBBS, PhD, MSc (2) · Donald B Kohn, MD (2) · Joseph Rossano, MD (2) · Agnieszka Czechowicz, MD (1) · Donald Kohn, MD (1) · Julian Sevilla Navarro, MD, PhD (1) · Julian Sevilla, MD, PhD (1) · Margaret MacMillan, MD, MSc (1) · Maria Chitty-Lopez, MD (1) · Matthew Taylor, MD, PhD (1) · Rajni Agarwal, MD (1)
PMID 40305711, PMID 36288619, PMID 37979797, PMID 39302117, PMID 39556016, PMID 39642902, PMID 40118805
2018 (1)