Not applicableStudy completedNCT00756821What this trial is testingA Pilot Study of Biomarkers for Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy Carelon Research 130
Large-scale testing (Phase 3)Active Not RecruitingNCT04042025What this trial is testingLong-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioiWho this might be right forSpinal Muscular Atrophy Type ISpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III+1 more Novartis Gene Therapies 85
Not applicableStudy completedNCT04193085What this trial is testingWearable Technology to Assess Gait Function in SMA and DMDWho this might be right forSpinal Muscular Atrophy Type 3Duchenne Muscular Dystrophy Columbia University 39
Testing effectiveness (Phase 2)Ended earlyNCT00439569What this trial is testingClinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or IIIWho this might be right forSpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type III Westat 9
Not applicableStudy completedNCT00466349What this trial is testingInternational SMA Patient RegistryWho this might be right forMuscular Atrophy, Spinal Indiana University School of Medicine 3,000
Large-scale testing (Phase 3)Active Not RecruitingNCT05626855What this trial is testingLong-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of ApitegromabWho this might be right forSpinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+7 more Scholar Rock, Inc. 238
Not applicableStudy completedNCT05430113What this trial is testingSpinal Cord Stimulation in Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 4 Marco Capogrosso 3
Testing effectiveness (Phase 2)Study completedNCT02227823What this trial is testingSafety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3Who this might be right forSpinal Muscular Atrophy Type 3 Centre Hospitalier Régional de la Citadelle 4
Not applicableLooking for participantsNCT06562283What this trial is testingEvaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular AtrophyWho this might be right forSpinal AmyotrophyInfantile Spinal Muscular AtrophyJuvenile Spinal Muscular Atrophy Centre Hospitalier Universitaire de Saint Etienne 80
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
Not applicableActive Not RecruitingNCT03709784What this trial is testingSpinraza in Adult Spinal Muscular AtrophyWho this might be right forSpinal Muscular AtrophySpinal Muscular Atrophy Type IISpinal Muscular Atrophy Type 3 Washington University School of Medicine 148
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 completedNCT02895789What this trial is testingOxidative Capacity and Exercise Tolerance in Ambulatory SMAWho this might be right forSpinal Muscular Atrophy Type 3Mitochondrial Myopathy Columbia University 42
Not applicableUnknownNCT05272969What this trial is testingPompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe DiseaseWho this might be right forPompe Disease (Late-onset)Inclusion Body MyositisSpinal Muscular Atrophy Type 3+1 more LMU Klinikum 95
Not applicableStudy completedNCT04404764What this trial is testingCharacterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational StudyWho this might be right forSpinal Muscular Atrophy Hospital Israelita Albert Einstein 155
Not applicableLooking for participantsNCT06839469What this trial is testingEstablishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular DisordersWho this might be right forSpinal Muscular Atrophy Type 3Duchenne Muscular Dystrophy (DMD) Columbia University 106
Not applicableUnknownNCT06137612What this trial is testingSpinal Cord Gray Matter Imaging in Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy Type IIISpinal Muscular Atrophy Type II University Hospital, Basel, Switzerland 52
Testing effectiveness (Phase 2)Study completedNCT03921528What this trial is testingAn Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular AtrophyWho this might be right forSpinal Muscular AtrophySpinal Muscular Atrophy Type 3Spinal Muscular Atrophy Type 2+6 more Scholar Rock, Inc. 58
Testing effectiveness (Phase 2)Study completedNCT03781479What this trial is testingControlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 PatientsWho this might be right forMuscular Atrophy, Spinal Catalyst Pharmaceuticals, Inc. 13
Not applicableUnknownNCT02831296What this trial is testingProspective Evaluation of Infants With Spinal Muscular Atrophy:Who this might be right forSpinal Muscular Atrophy Massachusetts General Hospital 1,000