1 active not recruiting, 1 completed, 1 enrolling by invitation, 1 unknown. 2 Phase 1, 2 Phase 2, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 2 | Active not recruiting 1, Unknown 1 |
| Phase 2 | 2 | Active not recruiting 1, Unknown 1 |
| Not applicable | 1 | Completed 1 |
Follow LGMD: new trials by email Literature Review of LGMD trials Research Report on LGMD
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 |
|---|---|---|---|---|---|---|
| Evaluating Safety and Efficacy of Autologous Gene-edited Muscle Stem Cells (GenPHSats-bASKet) GenPHSat injection (Safety), GenPHSat injection (Efficacy), Muscle Biopsy (Safety) - LGMD | Phase 1 / Phase 2 | Unknown | Simone Spuler, MD | 6 | 2024-07-01 | 2023-08-03 |
| The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder Shoulder orthosis - Muscular Dystrophy, Duchenne, Orthotic Devices | Not applicable | Completed | Seoul National University Hospital | 8 | 2024-04-20 | 2026-09-03 |
| A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) AB-1003 dose level 1, AB-1003 dose level 2, Placebo - Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2 | Phase 1 / Phase 2 | Active not recruiting | AskBio Inc | 9 | 2023-05-15 | 2026-08-26 |
| A Multicenter Phenotype-Genotype Analysis of LGMD Patients in China Electromyography, IDEAL MRI - LGMD, LGMDR2 | Enrolling by invitation | Huashan Hospital | 450 | 2021-07-07 | 2023-10-24 |
Limb-girdle muscular dystrophy type 2A (2) · Amyotrophic Lateral Sclerosis (1) · Limb-girdle muscular dystrophy, type 2B (1) · Muscular Atrophy, Spinal (1) · Muscular Dystrophies (1) · Muscular Dystrophies, Limb-Girdle (1) · Muscular Dystrophy, Duchenne (1) · Muscular Dystrophy, Facioscapulohumeral (1) · Muscular Dystrophy, Limb-Girdle, Type 2I (1) · Neuromuscular Diseases (1) · Spinal Cord Injuries (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Muscular Diseases (2) · Muscular Disorders, Atrophic (2) · Musculoskeletal Diseases (2) · Nervous System Diseases (2) · Central Nervous System Diseases (1) · Genetic Diseases, X-Linked (1) · Metabolic Diseases (1) · Motor Neuron Disease (1) · Muscular Dystrophies (1) · Neurodegenerative Diseases (1) · Neuromuscular Diseases (1) · Nutritional and Metabolic Diseases (1) · Proteostasis Deficiencies (1)
Electromyography (1) · Safety (1)
AskBio Inc (1) · Huashan Hospital (1) · Seoul National University Hospital (1) · Simone Spuler, MD (1)
China (1) · South Korea (1) · United States (1)
Chongbo Zhao, PhD (1) · Simone Spuler, Prof Dr med (1) · Woo Hyung Lee, MD, PhD (1)