Rocket Pharmaceuticals Inc.
Rocket Pharmaceuticals Inc.
Explore linked clinical trials.10 results ยท Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease | Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Danon disease, Hypertrophy, Hypertrophy, Left Ventricular, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, X-linked disease, aortic valve disorder, aortic valve stenosis, cardiomyopathy, cardiovascular disorder, disorder of glycogen metabolism, heart disorder, heart valve disorder, hereditary disease, hypertrophic cardiomyopathy, inborn carbohydrate metabolic disorder, inborn errors of metabolism, intellectual disability, lysosomal storage disease, metabolic disease, nervous system disorder, subvalvular aortic stenosis | Recruiting | Phase 2 | 2026-10-06 | NCT06092034 | |
| A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy | Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dilatation, Pathologic, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, heart failure, hereditary disease, laminopathy | Recruiting | Phase 1 | 2026-10-01 | NCT07137338 | |
| Danon Disease Natural History Study | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Danon disease, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, X-linked disease, cardiomyopathy, cardiovascular disorder, disorder of glycogen metabolism, heart disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, intellectual disability, metabolic disease, nervous system disorder | Recruiting | Not stated | 2026-07-28 | NCT06214507 | |
| A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM) | Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Death, Sudden, Cardiac, Pathologic Processes, Pathological Conditions, Signs and Symptoms, arrhythmogenic right ventricular cardiomyopathy, cardiac arrest, cardiomyopathy, cardiovascular disorder, congenital heart disease, heart disorder | Recruiting | Phase 1 | 2026-04-16 | NCT05885412 | |
| Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102 | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Hemic and Lymphatic Diseases, Nutritional and Metabolic Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, metabolic disease | Enrolling by invitation | Not stated | 2026-04-14 | NCT07527975 | |
| Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy | Bone Marrow Failure Disorders, Congenital Bone Marrow Failure Syndromes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Fanconi anemia, Fanconi anemia complementation group A, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Male Urogenital Diseases, Nutritional and Metabolic Diseases, Urogenital Diseases, anemia, aplastic anemia, bone marrow disorder, hematologic disorder, hereditary disease, inherited aplastic anemia, kidney disorder, metabolic disease, renal tubular transport disease, urinary system disorder | Active, not recruiting | Not stated | 2026-04-13 | NCT04437771 | |
| BAG3-DCM Natural History Study | Cardiomegaly, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, cardiomyopathy, cardiovascular disorder, dilated cardiomyopathy, heart disorder, hereditary disease, laminopathy | Not yet recruiting | Not stated | 2026-03-20 | NCT07486752 | |
| PKP2-ACM Natural History Study | Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, arrhythmogenic right ventricular cardiomyopathy, cardiomyopathy, cardiovascular disorder, congenital heart disease, heart disorder, hereditary disease | Recruiting | Not stated | 2026-02-27 | NCT06644742 | |
| Gene Therapy for Fanconi Anemia, Complementation Group A | Bone Marrow Failure Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fanconi anemia complementation group A, Fanconi renotubular syndrome, Female Urogenital Diseases, Female Urogenital Diseases and Pregnancy Complications, Hemic and Lymphatic Diseases, Male Urogenital Diseases, Urogenital Diseases, anemia, bone marrow disorder, hematologic disorder, hereditary disease, kidney disorder, renal tubular transport disease, urinary system disorder | Active, not recruiting | Phase 2 | 2025-12-22 | NCT04248439 | |
| Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) | leukocyte adhesion deficiency, leukocyte adhesion deficiency 1 | Active, not recruiting | Not stated | 2025-12-18 | NCT06282432 |