Early research (Phase 1)Study completedNCT02122952What this trial is testingGene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1Who this might be right forSpinal Muscular Atrophy 1 Novartis Gene Therapies 15
Not applicableActive Not RecruitingNCT05475691What this trial is testingLongitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin AmericaWho this might be right forSpinal Muscular Atrophy Hospital Israelita Albert Einstein 361
Not applicableEnded earlyNCT02550691What this trial is testingIdentification of a Biomarker Associated With Cis-duplication of the SMN1 GeneWho this might be right forSpinal Muscular Atrophy University Hospital, Rouen 48
Not applicableStudy completedNCT01862042What this trial is testingPalliative Care in Spinal Muscular Atrophy (SMA) 1Who this might be right forSpinal Muscular Atrophy 1 Assistance Publique - Hôpitaux de Paris 39
Not applicableEnrolling By InvitationNCT06256887What this trial is testingSleep Spindles Organization as an Early Neural Marker of Neuromotor OutcomeWho this might be right forEEG Sleep SpindlesMotor OutcomeCerebral Palsy+1 more IRCCS Fondazione Stella Maris 80
Not applicableStudy completedNCT00466349What this trial is testingInternational SMA Patient RegistryWho this might be right forMuscular Atrophy, Spinal Indiana University School of Medicine 3,000
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)Study completedNCT00568698What this trial is testingA Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy PatientsWho this might be right forMuscular Atrophy, Spinal Stanford University 29
Not applicableEnded earlyNCT01547871What this trial is testingInfants With Spinal Muscular Atrophy Type IWho this might be right forSpinal Muscular Atrophy National Institute of Neurological Disorders and Stroke (NINDS) 4
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
Not applicableNot Yet RecruitingNCT07208903What this trial is testingPsychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine RegionsWho this might be right forSpinal Muscular Atrophy (SMA)Spinal Muscular Atrophy Type I University Hospital, Strasbourg, France 36
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
Testing effectiveness (Phase 2)Enrolling By InvitationNCT05614531What this trial is testingClinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1Who this might be right forSpinal Muscular Atrophy Type I Hangzhou Jiayin Biotech Ltd 12
Not applicableActive Not RecruitingNCT03421977What this trial is testingLong-Term Follow-up Study for Patients From AVXS-101-CL-101Who this might be right forSpinal Muscular Atrophy 1 Novartis Gene Therapies 13
Not applicableStudy completedNCT02061189What this trial is testingPilot Study of an Innovative Physiotherapy in Patients With Infantile Spinal Muscular Atrophy (SMA)Who this might be right forInfantile Spinal Muscular Atrophy of Type 2 or 3 Assistance Publique - Hôpitaux de Paris 19
Not applicableLooking for participantsNCT05954455What this trial is testingExploring Bulbar Function, Speech And Communication Development in SMA Type 1Who this might be right forSMA1 Institute of Child Health 30
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07070999What this trial is testingStudy of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1Who this might be right forSpinal Muscular Atrophy Type I Gemma Biotherapeutics 22
Not applicableStudy completedNCT05715749What this trial is testingBody Weight Support Harness System in Spinal Muscular AtrophyWho this might be right forSpinal Muscular Atrophy Type ISpinal Muscular Atrophy Type II Nationwide Children's Hospital 33
Testing effectiveness (Phase 2)Ended earlyNCT00439218What this trial is testingClinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type IWho this might be right forSpinal Muscular Atrophy Type I Westat 5
Testing effectiveness (Phase 2)UnknownNCT02855112What this trial is testingAllogeneic Adipose Derived Stem Cells for Werdnig Hoffman PatientsWho this might be right forInfantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman] Tehran University of Medical Sciences 10