9 completed, 4 unknown, 3 recruiting, 2 active not recruiting. 18 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 4 | 18 | Completed 9, Unknown 4, Recruiting 3, Active not recruiting 2 |
Follow Motor Neuron Disease: new trials by email Literature Review of Motor Neuron Disease trials Research Report on Motor Neuron Disease
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 |
|---|---|---|---|---|---|---|
| ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM) Onasemnogene Abeparvovec-brve - Spinal Muscular Atrophy | Phase 4 | Recruiting | Novartis Pharmaceuticals | 36 | 2026-08-27 | 2026-10-29 |
| A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS) Tofersen - Amyotrophic Lateral Sclerosis | Phase 4 | Active not recruiting | Biogen | 12 | 2025-12-11 | 2026-08-13 |
| A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy Risdiplam - Muscular Atrophy, Spinal | Phase 4 | Active not recruiting | Hoffmann-La Roche | 19 | 2024-08-14 | 2026-10-22 |
| Effects of L-Carnitine in Amyotrophic Lateral Sclerosis Patients With CHCHD10 Mutations L-Carnitine Injection，1000mg once daily - Amyotrophic Lateral Sclerosis (ALS) | Phase 4 | Completed | ChaodongWang | 4 | 2024-07-18 | 2025-04-08 |
| A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy Risdiplam - Muscular Atrophy, Spinal | Phase 4 | Recruiting | Hoffmann-La Roche | 28 | 2024-05-30 | 2026-10-30 |
| Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients FB1006, Placebo - Sporadic and Familial Amyotrophic Lateral Sclerosis | Phase 4 | Recruiting | Peking University Third Hospital | 64 | 2023-01-18 | 2023-11-22 |
| Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen Risdiplam - Spinal Muscular Atrophy | Phase 4 | Unknown | Clinic for Special Children | 10 | 2022-11-06 | 2024-02-07 |
| Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) Risdiplam - Spinal Muscular Atrophy | Phase 4 | Completed | Genentech, Inc. | 403 | 2022-03-29 | 2026-10-22 |
| Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) OAV101 - Muscular Atrophy, Spinal | Phase 4 | Completed | Novartis Pharmaceuticals | 16 | 2021-11-04 | 2024-10-09 |
| EMERALD TRIAL Open Label Extension Study MediCabilis CBD oil - Amyotrophic Lateral Sclerosis, Cannabis | Phase 4 | Unknown | Gold Coast Hospital and Health Service | 7 | 2021-05-17 | 2023-04-03 |
| A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND) Nusinersen - Muscular Atrophy, Spinal | Phase 4 | Completed | Biogen | 46 | 2021-01-04 | 2026-10-30 |
| Paracetamol Study in Patients With Low Muscle Mass Paracetamol 120Mg/5mL Oral Suspension - SMA II, Cerebral Palsy | Phase 4 | Unknown | Mette Cathrine Oerngreen | 48 | 2019-02-18 | 2024-02-22 |
| Mexiletine for the Treatment of Muscle Cramps in ALS Mexiletine, Placebo - Muscle Cramps in Amyotrophic Lateral Sclerosis | Phase 4 | Completed | Bjorn Oskarsson, MD | 23 | 2013-05-01 | 2017-08-28 |
| Flu Vaccine Study in Neuromuscular Patients 2011 2011-2012 seasonal flu vaccine Subcutaneous, 2011-2012 seasonal flu vaccine Intramuscular - Duchenne Muscular Dystrophy, Spinal Muscular Atrophy | Phase 4 | Completed | Children's Hospital Medical Center, Cincinnati | 22 | 2011-08-01 | 2020-10-19 |
| Effect of Goserelin (Zoladex®) in Spinal and Bulbar Muscular Atrophy Goserelin, Electrophysiologic study, tissue biopsy - Spinobulbar Muscular Atrophy, Kennedy's Disease | Phase 4 | Completed | Ramathibodi Hospital | 10 | 2008-04-01 | 2015-05-21 |
| Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers, Non invasive ventilation - Amyotrophic Lateral Sclerosis, Chronic Respiratory Failure | Phase 4 | Unknown | Fondazione Salvatore Maugeri | 50 | 2008-01-01 | 2010-03-29 |
| Feasibility of Telesurveillance and Home Cough Assistance for Amyotrophic Lateral Patients (ALS) telesurveillance - Amyotrophic Lateral Sclerosis | Phase 4 | Completed | Fondazione Salvatore Maugeri | 40 | 2007-10-01 | 2016-02-04 |
| CARE Canadian ALS Riluzole Evaluation Riluzole - Amyotrophic Lateral Sclerosis | Phase 4 | Completed | Sanofi | 414 | 2001-01-01 | 2007-10-11 |
Amyotrophic Lateral Sclerosis (8) · Muscular Atrophy, Spinal (8) · Bulbo-Spinal Atrophy, X-Linked (2) · Cerebral Palsy (1) · Influenza, Human (1) · Marijuana Abuse (1) · Muscle Cramp (1) · Muscular Atrophy (1) · Muscular Diseases (1) · Muscular Dystrophy, Duchenne (1) · Spinal Muscular Atrophies of Childhood (1)
Central Nervous System Diseases (18) · Nervous System Diseases (18) · Neurodegenerative Diseases (18) · Neuromuscular Diseases (18) · Spinal Cord Diseases (18) · Metabolic Diseases (8) · Nutritional and Metabolic Diseases (8) · Proteostasis Deficiencies (8) · TDP-43 Proteinopathies (8) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (4) · Genetic Diseases, Inborn (4) · Genetic Diseases, X-Linked (3) · Heredodegenerative Disorders, Nervous System (3) · Musculoskeletal Diseases (3)
Risdiplam (4) · Acetaminophen (1) · Goserelin (1) · Influenza Vaccines (1) · Mexiletine (1) · Noninvasive Ventilation (1) · Riluzole (1) · Sugars (1) · Suspensions (1) · Telemedicine (1) · Zolgensma (1) · nusinersen (1) · tofersen (1)
Biogen (2) · Fondazione Salvatore Maugeri (2) · Hoffmann-La Roche (2) · Novartis Pharmaceuticals (2) · Bjorn Oskarsson, MD (1) · ChaodongWang (1) · Children's Hospital Medical Center, Cincinnati (1) · Clinic for Special Children (1) · Genentech, Inc. (1) · Gold Coast Hospital and Health Service (1) · Mette Cathrine Oerngreen (1) · Peking University Third Hospital (1) · Ramathibodi Hospital (1) · Sanofi (1)
United States (7) · China (3) · Germany (3) · Italy (3) · Israel (2) · Poland (2) · United Kingdom (2) · Argentina (1) · Australia (1) · Brazil (1) · Canada (1) · Denmark (1) · Puerto Rico (1) · Qatar (1) · Spain (1)
Bjorn Oskarsson, MD (1) · Brenda Wong, MD (1) · Fan DongSheng (1) · Laurent-Didier Jacobs (1) · Stefano Nava (1) · Stefano Nava, MD (1) · Thanyachau Sura, M.D., MRCP (1)
PMID 20407305, PMID 24491372, PMID 40956616
2023 (4), 2018 (1)