4 recruiting, 2 completed, 1 unknown. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Completed 1 |
| Phase 2 | 1 | Recruiting 1 |
Follow Glycogen Storage Disease Type IIb: new trials by email Literature Review of Glycogen Storage Disease Type IIb trials Research Report on Glycogen Storage Disease Type IIb
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 |
|---|---|---|---|---|---|---|
| Rare Glycogen Storage Diseases Natural History Study No intervention - Glycogen Storage Disease, GSD Type 0A | Recruiting | Duke University | 200 | 2024-12-23 | 2026-01-07 | |
| 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 |
| Natural History of Danon Disease Heart Transplant, Cardiac Assistive Device - Danon Disease | Completed | Rocket Pharmaceuticals Inc. | 59 | 2022-09-26 | 2023-11-21 | |
| 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 |
| The Natural History of Danon Disease Danon Disease | Unknown | University of California, San Diego | 200 | 2018-11-30 | 2022-05-12 | |
| Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques Danon Disease, Fabry Disease | Recruiting | Chinese Academy of Medical Sciences, Fuwai Hospital | 1000 | 2010-01-01 | 2026-01-21 |
Cardiomyopathies (3) · Cardiomyopathy, Hypertrophic (2) · Glycogen Storage Disease (2) · Amyloid Neuropathies, Familial (1) · Dimauro disease (1) · Disease (1) · Fabry Disease (1) · Glycogen Storage Disease Type VII (1) · Glycogen Storage Disease XII (1) · Glycogen Storage Disease XIII (1) · Hypertrophy, Left Ventricular (1) · Lysosomal Storage Diseases (1) · Noonan Syndrome (1)
Carbohydrate Metabolism, Inborn Errors (7) · Cardiovascular Diseases (7) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (7) · Genetic Diseases, Inborn (7) · Genetic Diseases, X-Linked (7) · Heart Diseases (7) · Intellectual Disability (7) · Metabolic Diseases (7) · Metabolism, Inborn Errors (7) · Nervous System Diseases (7) · Neurobehavioral Manifestations (7) · Neurologic Manifestations (7) · Nutritional and Metabolic Diseases (7) · X-Linked Intellectual Disability (7) · Glycogen Storage Disease (5)
Rocket Pharmaceuticals Inc. (4) · Chinese Academy of Medical Sciences, Fuwai Hospital (1) · Duke University (1) · University of California, San Diego (1)
United States (5) · Italy (3) · Germany (2) · Spain (2) · United Kingdom (2) · Australia (1) · China (1) · Czechia (1) · France (1) · Ireland (1) · Israel (1)
Barry Greenberg, MD (2) · Joseph Rossano, MD (2) · Matthew Taylor, MD, PhD (1) · Priya Kishnani, M.D. (1)
PMID 32499120, PMID 36288619, PMID 39290522, PMID 39302117, PMID 39344930, PMID 39556016, PMID 39659467, PMID 39700428, PMID 40118805