Danon disease
Danon disease
Explore linked clinical trials. Standard reference: MONDO:00102814 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 | |
| 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 | |
| Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques | Amyloid Neuropathies, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Cerebral Small Vessel Diseases, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Danon disease, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Musculoskeletal Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Noonan syndrome, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, X-linked disease, amyloidosis, brain disorder, cardiac sarcoidosis, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, congenital heart disease, connective tissue disorder, disease, disorder of glycogen metabolism, familial amyloid neuropathy, heart disorder, hereditary amyloidosis, hereditary disease, idiopathic cardiomyopathy, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, peripheral nervous system disorder, proteostasis deficiencies, sphingolipidosis, vascular disorder | Recruiting | Not stated | 2026-01-21 | NCT07336394 | |
| Rare Glycogen Storage Diseases Natural History Study | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Danon disease, Muscular Disorders, Atrophic, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, X-linked disease, cardiomyopathy, cardiovascular disorder, disorder of glycogen metabolism, glycogen storage disease VII, glycogen storage disease due to aldolase A deficiency, glycogen storage disease due to muscle and heart glycogen synthase deficiency, glycogen storage disease due to muscle beta-enolase deficiency, glycogen storage disease due to phosphoglycerate mutase deficiency, glycogen storage disorder due to hepatic glycogen synthase deficiency, heart disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, intellectual disability, metabolic disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease, polyglucosan body myopathy 1 with or without immunodeficiency, polyglucosan body myopathy type 2 | Recruiting | Not stated | 2026-01-07 | NCT06795152 |