Recruiting
A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec
NCT07444450· Phase 3· Biogen· Source
Not yet recruiting
Colchicine Effect on Amyotrophic Lateral Sclerosis Patients
NCT07813858· Phase 2· Azienda Ospedaliero-Universitaria di Modena· Source
Not yet recruiting
A RANDOMIZED, PLACEBO-CONTROLLED, MULTICENTER, CLINICAL TRIAL OF COLCHICINE IN AMYOTROPHIC LATERAL SCLEROSIS (CO-ALS II)
2026-526460-20-00· Phase 2· Azienda Ospedaliero Universitaria Di Modena· Source
Not yet recruiting
Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)
NCT07682129· Phase 2· Entrada Therapeutics, Inc.· Source
Recruiting
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping
2025-523087-20-00· Phase 3· Avidity Biosciences Inc.· Source
Recruiting
Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS (PREVAiLS)
2025-524002-16-00· Phase 3· Ferrer Internacional S.A.· Source
Recruiting
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
NCT07221669· Phase 3· Biogen· Source
Enrolling by invitation
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
NCT07573631· Phase 2· BioMarin Pharmaceutical· Source
Recruiting
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
2024-520130-29-00· Phase 2· Scholar Rock Inc.· Source
Recruiting
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
NCT07160634· Phase 3· Solid Biosciences Inc.· Source
Recruiting
Usnoflast Neuromuscular Investigation for Treatment Efficacy in Amyotrophic Lateral Sclerosis
NCT07023835· Phase 2· Zydus Therapeutics Inc.· Source
Recruiting
A Study to Assess the Efficacy and Safety of Empasiprubart in Adults With CIDP
NCT07091630· Phase 3· argenx· Source
Recruiting
Respiratory Progression in Adults With Duchenne Muscular Dystrophy.
NCT07879989· Istituto Giannina Gaslini· Source
Recruiting
A Study to Evaluate the Efficacy and Safety of DNTH103 in Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CAPTIVATE)
2024-517529-26-00· Phase 3· Dianthus Therapeutics Inc.· Source
Recruiting
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
NCT07037862· Phase 1/2· Entrada Therapeutics, Inc.· Source
Recruiting
Trial on the Biological and Clinical Effects of Acetyl-L-carnitine in ALS
NCT06126315· Phase 2/3· Mario Negri Institute for Pharmacological Research· Source
Recruiting
Imeroprubart in Adult Participants With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
NCT07032662· Phase 2· Immunovant Sciences GmbH· Source
Recruiting
A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old
2024-511823-32-00· Phase 2· Italfarmaco S.p.A.· Source
Recruiting
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
NCT05933057· Phase 3· Italfarmaco· Source
Active, not recruiting
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
NCT05626855· Phase 3· Scholar Rock, Inc.· Source
Recruiting
Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments
NCT05768048· Fondazione Policlinico Universitario Agostino Gemelli IRCCS· Source
Active, not recruiting
A Study to Learn About the Safety of BIIB115 Injections and How BIIB115 is Processed in the Bodies of Healthy Adult Male Volunteers and of Pediatric Participants With Spinal Muscular Atrophy Who Previously Took Onasemnogene Abeparvovec
NCT05575011· Phase 1· Biogen· Source
Active, not recruiting
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
NCT05524883· Phase 1/2· Dyne Therapeutics· Source
Active, not recruiting
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
NCT05337553· Phase 3· Biohaven Pharmaceuticals, Inc.· Source
Active, not recruiting
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
NCT05067790· Phase 3· Biogen· Source
Recruiting
Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients
NCT03127267· Phase 3· AB Science· Source
Recruiting
Natural History of SMA
NCT05755451· Fondazione Policlinico Universitario Agostino Gemelli IRCCS· Source
Recruiting
Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past
2023-504520-26-00· Phase 3· Italfarmaco S.p.A.· Source