Not applicableStudy completedNCT01754441What this trial is testingMechanisms of Cell Death in Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy Nemours Children's Clinic 25
Testing effectiveness (Phase 2)Looking for participantsNCT06421831What this trial is testingEvaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 PatientsWho this might be right forSpinal Muscular Atrophy Type 3 GeneCradle Inc 21
Not applicableStudy completedNCT04813601What this trial is testingRehabilitative Effect of the Use of a Gait Exoskeleton in Patients With Neuromuscular Disease or Cerebral PalsyWho this might be right forNeuromuscular DiseasesCerebral PalsySMA II MarsiBionics 10
Testing effectiveness (Phase 2)Ended earlyNCT03196375What this trial is testingAssess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.Who this might be right forMotor Neuron Disease Flex Pharma, Inc. 54
Testing effectiveness (Phase 2)Study completedNCT01703988What this trial is testingAn Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy Biogen 34
Not applicableLooking for participantsNCT05663008What this trial is testingImpairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised DiagnosisWho this might be right forALS (Amyotrophic Lateral Sclerosis)Postpoliomyelitis SyndromeSpinal Muscular Atrophy University of Dublin, Trinity College 400
Not applicableLooking for participantsNCT07223489What this trial is testingDiagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health SystemWho this might be right forSpinal Muscular Atrophy (SMA) Medstar Health Research Institute 200
Not applicableWithdrawnNCT01663584What this trial is testingMulti-disease Carrier Screening Test ValidationWho this might be right forSpinal Muscular Atrophy (SMA)Carrier ScreeningGenetic Testing Natera, Inc.
Not applicableLooking for participantsNCT03217578What this trial is testingNeonatal Spinal Muscular Atrophy (SMA) ScreeningWho this might be right forSpinal Muscular Atrophy Kaohsiung Medical University Chung-Ho Memorial Hospital 250,000
Not applicableStudy completedNCT07378943What this trial is testingDosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying TherapiesWho this might be right forMuscular Atrophy, Spinal Novartis Pharmaceuticals 4,114
Not applicableStudy completedNCT02574390What this trial is testingAnswer ALS: Individualized Initiative for ALS DiscoveryWho this might be right forAmyotrophic Lateral SclerosisPrimary Lateral SclerosisFlail Arm ALS+5 more Johns Hopkins University 1,049
Testing effectiveness (Phase 2)Looking for participantsNCT07287982What this trial is testingAssess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy (SMA) argenx 60
Early research (Phase 1)Study completedNCT02633709What this trial is testingInvestigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy VolunteersWho this might be right forSpinal Muscular Atrophy Hoffmann-La Roche 33
Testing effectiveness (Phase 2)Ended earlyNCT03819660What this trial is testingLong Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3Who this might be right forSpinal Muscular Atrophy Type 3 Catalyst Pharmaceuticals, Inc. 13
Large-scale testing (Phase 3)Study completedNCT05156320What this trial is testingEfficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or RisdiplamWho this might be right forSpinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+7 more Scholar Rock, Inc. 188
Not applicableUnknownNCT03520179What this trial is testingImproving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA)Who this might be right forSpinal Muscular Atrophy Great Ormond Street Hospital for Children NHS Foundation Trust 600
Post-approval studies (Phase 4)Study completedNCT04488133What this trial is testingLearn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)Who this might be right forMuscular Atrophy, Spinal Biogen 46
Not applicableLooking for participantsNCT06756633What this trial is testingRespiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular DiseasesWho this might be right forNeuromuscular Diseases in ChildrenRespiratory Function ImpairedRespiratory Insufficiency+3 more Lokman Hekim University 42
Not applicableStudy completedNCT02156141What this trial is testingHigh Intensity Training in Patients With Spinal and Bulbar Muscular AtrophyWho this might be right forSpinal and Bulbar Muscular AtrophyHealthy Subjects Karen Brorup Heje Pedersen 10
Not applicableStudy completedNCT03560661What this trial is testingAcoustic and Perceptual Markers of Dysarthria in Amyotrophic Lateral Sclerosis (ALS)Who this might be right forAmyotrophic Lateral SclerosisPrimary Lateral SclerosisKennedy's Disease Assistance Publique - Hôpitaux de Paris 71