13 recruiting, 2 active not recruiting, 2 completed, 1 not yet recruiting. 8 Not applicable, 3 Phase 3, 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 8 | Recruiting 5, Completed 2, Active not recruiting 1 |
| Phase 3 | 3 | Recruiting 3 |
| Phase 1 | 1 | Active not recruiting 1 |
| Phase 2 | 1 | Active not recruiting 1 |
Follow Neuromuscular Diseases (NMD): new trials by email Literature Review of Neuromuscular Diseases (NMD) trials Research Report on Neuromuscular Diseases (NMD)
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 |
|---|---|---|---|---|---|---|
| Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders Standard of Care (Investigator Choice) - ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy | Recruiting | Ambulanzpartner Soziotechnologie APST GmbH | 2000 | 2026-10-01 | 2026-10-28 | |
| 3D Hand Orthosis Trial 3D-printed hand orthosis (intervention), Conventional hand orthosis (control) - Hand Injuries and Disorders, Neurological Conditions | Not applicable | Recruiting | Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) | 100 | 2026-09-14 | 2026-09-30 |
| A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping AOC 1044, Placebo - Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy) | Phase 3 | Recruiting | Avidity Biosciences, Inc. | 70 | 2026-09-01 | 2026-10-13 |
| Clinical Investigation to Validate the Safety and Performance of the ABLE Kids Device in Pediatric Patients With Neurological Gait Disorders in a Clinical Setting ABLE Kids - Gait Impairment, Cerebral Palsy (CP) | Not applicable | Recruiting | ABLE Human Motion S.L. | 15 | 2026-09-01 | 2026-09-08 |
| Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) Zeleciment Rostudirsen (DYNE-251), Placebo - Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne | Phase 3 | Recruiting | Dyne Therapeutics | 90 | 2026-06-01 | 2026-05-27 |
| Northwestern Neuromuscular Disease Biorepository Neuromuscular Diseases (NMD) | Recruiting | Northwestern University | 2500 | 2026-05-01 | 2026-05-26 | |
| Invasive Home Ventilation in Denmark Neuromuscular Diseases (NMD), ALS (Amyotrophic Lateral Sclerosis) | Recruiting | Copenhagen University Hospital, Rigshospitalet | 450 | 2026-04-13 | 2026-04-20 | |
| Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease Whole-body Electrical Muscle Stimulation Exercise - Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis | Not applicable | Recruiting | University of Missouri-Columbia | 50 | 2026-03-10 | 2026-03-17 |
| Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study Functional Surgery plus Rehabilitation, Physical Exercise - Charcot Marie Tooth Disease (CMT), Pes Cavovarus | Not yet recruiting | Peking University Third Hospital | 200 | 2026-02-11 | 2026-02-25 | |
| Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy? Routine Clinical Follow-up and Pain Assessment - Duchenne Muscular Dystrophy (DMD), Chronic Pain | Recruiting | Antalya Training and Research Hospital | 30 | 2026-01-02 | 2026-10-23 | |
| EMMVIES : Walking Training at Home in VIrtual Reality for Children With Chronic Illnesses Impacting Physical Health EMMVIES program - Home-based Programs, EzyGain | Not applicable | Recruiting | University Hospital, Angers | 30 | 2025-12-24 | 2025-12-31 |
| Ultrasound Evaluation of Hematoma Risk After Needle EMG in Patient on DOAC Therapy Direct Acting Anticoagulant Adverse Reaction, Needle Injury | Recruiting | Masaryk University | 70 | 2025-09-01 | 2025-09-10 | |
| Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient VCA-894A - Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD) | Phase 1 / Phase 2 | Active not recruiting | Vanda Pharmaceuticals | 1 | 2025-05-13 | 2026-08-19 |
| Personalized Training for People With Rare Neuromuscular Disorders Exercise, Usual Care - Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT) | Not applicable | Recruiting | Oslo University Hospital | 120 | 2024-12-13 | 2026-06-18 |
| A Phase 3 Study of NTLA-2001 in ATTRv-PN nexiguran ziclumeran, Normal Saline as Placebo - Neuromuscular Disease, Neuromuscular Diseases (NMD) | Phase 3 | Recruiting | Intellia Therapeutics | 60 | 2024-11-22 | 2026-04-16 |
| Enhancement of Quality of Work And Life Preventive Participatory workplace intervention - Parkinson Disease, Hereditary Spastic Paraparesis | Not applicable | Active not recruiting | Radboud University Medical Center | 124 | 2024-10-08 | 2026-08-14 |
| Evaluation of Auto-EPAP for Management of Upper Airway Obstruction During Non-Invasive Ventilation Non-Invasive Ventilation - Obstructive Sleep Apnea, Obesity Hypoventilation Syndrome (OHS) | Not applicable | Completed | Breas Medical, Inc. | 23 | 2024-10-03 | 2026-01-28 |
| 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 |
Neuromuscular Diseases (18) · Muscular Dystrophy, Duchenne (5) · Amyotrophic Lateral Sclerosis (4) · Charcot-Marie-Tooth Disease (4) · Muscular Atrophy, Spinal (4) · Genetic Diseases, Inborn (3) · Muscular Dystrophy, Facioscapulohumeral (3) · Myotonic Dystrophy (3) · Nervous System Diseases (3) · Neurodegenerative Diseases (3) · Spinal Cord Injuries (3) · Cerebral Palsy (2) · Metabolic Diseases (2) · Motor Neuron Disease (2) · Muscular Dystrophies (2)
Nervous System Diseases (15) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Pathological Conditions, Signs and Symptoms (9) · Genetic Diseases, Inborn (8) · Muscular Diseases (8) · Musculoskeletal Diseases (8) · Neurodegenerative Diseases (8) · Heredodegenerative Disorders, Nervous System (7) · Nutritional and Metabolic Diseases (7) · Central Nervous System Diseases (6) · Congenital Abnormalities (6) · Muscular Disorders, Atrophic (6) · Nervous System Malformations (6) · Brain Diseases (5) · Hereditary Sensory and Motor Neuropathy (5)
Exercise (2) · Methods (1) · NTLA-2001 (1) · Noninvasive Ventilation (1) · Rehabilitation (1) · Saline Solution (1) · Standard of Care (1)
ABLE Human Motion S.L. (1) · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) (1) · Ambulanzpartner Soziotechnologie APST GmbH (1) · Antalya Training and Research Hospital (1) · Avidity Biosciences, Inc. (1) · Breas Medical, Inc. (1) · Copenhagen University Hospital, Rigshospitalet (1) · Dyne Therapeutics (1) · Intellia Therapeutics (1) · Masaryk University (1) · Northwestern University (1) · Oslo University Hospital (1) · Peking University Third Hospital (1) · Radboud University Medical Center (1) · Seoul National University Hospital (1)
United States (5) · Netherlands (3) · France (2) · Germany (2) · Spain (2) · Argentina (1) · Australia (1) · Belgium (1) · Brazil (1) · Czechia (1) · Denmark (1) · Italy (1) · Mexico (1) · Norway (1) · Singapore (1)
Elbrich Postma, PhD (1) · Eva Vlckova, MD (1) · Merel-Anne Brehm, PhD (1) · Woo Hyung Lee, MD, PhD (1)
2025 (1)