1 completed. 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Completed 1 |
Follow Danon Disease: new trials by email Literature Review of Danon Disease trials Research Report on Danon Disease
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 |
|---|---|---|---|---|---|---|
| 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 |
Cardiomyopathy, Hypertrophic (1) · Glycogen Storage Disease Type IIb (1)
Aortic Stenosis, Subvalvular (1) · Aortic Valve Disease (1) · Aortic Valve Stenosis (1) · Carbohydrate Metabolism, Inborn Errors (1) · Cardiomyopathies (1) · Cardiovascular Diseases (1) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1) · Genetic Diseases, Inborn (1) · Genetic Diseases, X-Linked (1) · Glycogen Storage Disease (1) · Heart Diseases (1) · Heart Valve Diseases (1) · Intellectual Disability (1) · Metabolic Diseases (1) · Metabolism, Inborn Errors (1)
Rocket Pharmaceuticals Inc. (1)
United States (1)
Barry Greenberg, MD (1) · Joseph Rossano, MD (1) · Matthew Taylor, MD, PhD (1)