Muscular Atrophy, Spinal: all 295 studies

Page 1 of 3. Open to participants first (78), then studies with results posted, then the rest; newest registration first within each.

• SMAdult - Monitoring of Movement on Spinal Muscular AtrophyRecruitingNCT07894185Not applicable
• Service Evaluation for Newborn Screening for Spinal Muscular AtrophyRecruitingNCT07888868
• Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMARecruitingNCT07883655
• An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)Not yet recruitingNCT07846696Phase 3
• An OLIP Study (Open Label Individual Patient) of Nusinersen in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With NusinersenNot yet recruitingNCT07838779Not applicable
• Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMARecruitingNCT07822711Not applicable
• A Registry of Cases of Spinal Muscular AtrophyRecruitingNCT07805278
• A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy PatientsRecruitingNCT07767890
• A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi ArabiaNot yet recruitingNCT07737756
• Spinal Muscular Atrophy and School TransitionsNot yet recruitingNCT07720518
• An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)RecruitingNCT07543003
• A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)RecruitingNCT07531719
• Adrenal& Bone Complications in Paediatric Patients Living With Spinal Muscular Atrophy (SMA)Not yet recruitingNCT07530926
• Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)RecruitingNCT07521930Not applicable
• HABIT-ILE + FST in Children With SMA: Preliminary EffectivenessRecruitingNCT07488338Not applicable
• Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular DiseaseRecruitingNCT07478172Not applicable
• Invasive Home Ventilation in DenmarkRecruitingNCT07467187
• 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)RecruitingNCT07448610Phase 4
• A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With RisdiplamRecruitingNCT07444476Phase 3
• 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 AbeparvovecRecruitingNCT07444450Phase 3
• Gait and Bone Health in SMARecruitingNCT07400198
• Neuroproprioceptive Equine-Assisted Physiotherapy for Spinal Muscular AtrophyNot yet recruitingNCT07336602Not applicable
• Long Read Analysis in Spinal Muscular Atrophy - LOREASIRecruitingNCT07332702Not applicable
• Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic VolunteersRecruitingNCT07321977Not applicable
• A Study to Assess 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 AtrophyRecruitingNCT07287982Phase 2
• Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn ScreeningRecruitingNCT07286565Not applicable
• CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in SpainRecruitingNCT07231549
• Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health SystemRecruitingNCT07223489
• 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)RecruitingNCT07221669Phase 3
• Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine RegionsNot yet recruitingNCT07208903
• Safety, Usability, and Effectiveness of a Gait Exoskeleton for Children and Adolescents With Neurodevelopmental Disorders.Not yet recruitingNCT07159360Not applicable
• The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Enrolling by invitationNCT07072676Not applicable
• A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular AtrophyRecruitingNCT07047144Phase 2
• Adult SMA Research and Clinical HubRecruitingNCT06978985
• Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular AtrophyRecruitingNCT06977269Not applicable
• Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase IIIRecruitingNCT06971094Phase 3
• Characterizing Perceived Physical Fatigability in Nusinersen-treated SMARecruitingNCT06955897
• Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular AtrophyEnrolling by invitationNCT06888661Early Phase 1
• Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular DisordersRecruitingNCT06839469
• Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular DiseasesRecruitingNCT06756633
• Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular DiseasesRecruitingNCT06666816
• Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular DisordersEnrolling by invitationNCT06605612
• A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMAEnrolling by invitationNCT06588803
• Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular AtrophyRecruitingNCT06562283Not applicable
• A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)RecruitingNCT06555419Phase 1
• Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed TherapiesRecruitingNCT06532474
• A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular AtrophyRecruitingNCT06396325Not applicable
• High-intensity Interval Training in Patients With Spinal Muscular AtrophyRecruitingNCT06368076Not applicable
• Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring TherapyRecruitingNCT06321965Not applicable
• Spinal Muscular Atrophy Neonatal Screening ProgramRecruitingNCT06310421
• Assessing the Fertility Status of Men With Spinal Muscular Atrophy (SMA)RecruitingNCT06194539
• Spinal Cord Gray Matter Imaging in Spinal Muscular AtrophyEnrolling by invitationNCT06137612
• A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With ZolgensmaRecruitingNCT06019637
• A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022RecruitingNCT05994950
• ALS/MND Natural History Study Data RepositoryRecruitingNCT05966038
• Motor Function and Activity of Daily Life in Spinal Muscular AtrophyNot yet recruitingNCT05931211
• Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMANot yet recruitingNCT05866939
• Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular AtrophyRecruitingNCT05866419Not applicable
• A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene TherapyRecruitingNCT05861986Phase 4
• Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation StudyNot yet recruitingNCT05839145Not applicable
• Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 PatientsRecruitingNCT05824169Phase 1/2
• A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their BabiesRecruitingNCT05789758
• MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular AtrophyRecruitingNCT05769465
• Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying TreatmentsRecruitingNCT05768048
• Natural History of SMARecruitingNCT05755451
• Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)RecruitingNCT05747261Phase 1/2
• Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised DiagnosisRecruitingNCT05663008
• Outpatient Rehabilitation Intervention for Young Children With SMARecruitingNCT05638750Not applicable
• Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1Enrolling by invitationNCT05614531Phase 1/2
• The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular AtrophyRecruitingNCT05544994Not applicable
• Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe DiseaseRecruitingNCT05272969
• Comprehensive Analysis Platform To Understand, Remedy and Eliminate ALSRecruitingNCT05204017
• UK SMA Patient RegistryRecruitingNCT04292574
• French Register of Patients With Spinal Muscular AtrophyRecruitingNCT04177134
• Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)RecruitingNCT04174157
• A Registered Cohort Study on SMARecruitingNCT04010604
• Clinical Procedures to Support Research in ALSRecruitingNCT03489278
• Neonatal Spinal Muscular Atrophy (SMA) ScreeningRecruitingNCT03217578
• Spinal Cord Stimulation in Spinal Muscular AtrophyCompletedNCT05430113Not applicableResults posted
• Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or RisdiplamCompletedNCT05386680Phase 3Results posted
• Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)CompletedNCT05089656Phase 3Results posted
• Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)CompletedNCT05073133Phase 4Results posted
• Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)CompletedNCT04851873Phase 3Results posted
• Study of Nusinersen (BIIB058) in Participants With Spinal Muscular AtrophyCompletedNCT04089566Phase 3Results posted
• A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy ParticipantsCompletedNCT03988907Phase 1Results posted
• An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular AtrophyCompletedNCT03921528Phase 2Results posted
• A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic FunctionCompletedNCT03920865Phase 1Results posted
• Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 CopiesCompletedNCT03837184Phase 3Results posted
• Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3TerminatedNCT03819660Phase 2Results posted
• Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 PatientsCompletedNCT03781479Phase 2Results posted
• A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyActive, not recruitingNCT03779334Phase 2Results posted
• Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2CompletedNCT03505099Phase 3Results posted
• Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular AtrophyTerminatedNCT03381729Phase 1Results posted
• Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1CompletedNCT03306277Phase 3Results posted
• Whole Body Vibration Therapy in Children With Spinal Muscular AtrophyTerminatedNCT03056144Not applicableResults posted
• A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular AtrophyCompletedNCT03032172Phase 2Results posted
• Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular AtrophyCompletedNCT02913482Phase 2Results posted
• A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) ParticipantsCompletedNCT02908685Phase 2Results posted
• A Study of CK-2127107 in Patients With Spinal Muscular AtrophyCompletedNCT02644668Phase 2Results posted
• A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)CompletedNCT02628743Phase 2Results posted

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