A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Recruiting · NCT06092034 · Registry updated 2026-10-06Clinical 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 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Rocket Pharmaceuticals Inc. | 10 |
6 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Boston Children's Hospital | Boston | United States | 343 |
| Children's Hospital of Philadelphia | Philadelphia | United States | 434 |
| German Heart Center Munich | Munich | Germany | 4 |
| Great Ormond Street Hospital & UCL Institute of Cardiovascular Science | London | United Kingdom | 1 |
| Meyer Children's Hospital | Florence | Italy | 1 |
| University of California, San Diego | La Jolla | United States | 25 |