Muscular Atrophy, Spinal: all 295 studies

Page 2 of 3. Open to participants first (78), then studies with results posted, then the rest; newest registration first within each.

• A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational StudiesCompletedNCT02594124Phase 3Results posted
• A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).TerminatedNCT02462759Phase 2Results posted
• A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyCompletedNCT02386553Phase 2Results posted
• A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)CompletedNCT02292537Phase 3Results posted
• An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)CompletedNCT02268552Phase 1/2Results posted
• WREX Outcome StudyCompletedNCT02218593Results posted
• A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular AtrophyTerminatedNCT02193074Phase 3Results posted
• Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1CompletedNCT02122952Phase 1Results posted
• A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)CompletedNCT01839656Phase 2Results posted
• Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of DevelopmentCompletedNCT01736553Results posted
• An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular AtrophyCompletedNCT01703988Phase 1/2Results posted
• Short and Long Term Treatment With 4-AP in Ambulatory SMA PatientsCompletedNCT01645787Phase 2/3Results posted
• Flu Vaccine Study in Neuromuscular Patients 2011CompletedNCT01422200Phase 4Results posted
• Progressive Strength Training in Spinal Muscular AtrophyCompletedNCT01233817Not applicableResults posted
• CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type ICompletedNCT00661453Phase 1/2Results posted
• A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy PatientsCompletedNCT00568802Phase 1/2Results posted
• A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy PatientsCompletedNCT00568698Phase 1/2Results posted
• Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular AtrophyCompletedNCT00528268Phase 1/2Results posted
• Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or IIITerminatedNCT00439569Phase 1/2Results posted
• Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type ITerminatedNCT00439218Phase 1/2Results posted
• Valproic Acid and Carnitine in Patients With Spinal Muscular AtrophyCompletedNCT00227266Phase 2Results posted
• A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular AtrophyActive, not recruitingNCT07617779Not applicable
• VRehab-SMA Phase 1.2Active, not recruitingNCT07578454Not applicable
• Effects of Developmental Support for Mothers of Infants With Spinal Muscular AtrophyCompletedNCT07547189Not applicable
• A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular AtrophyCompletedNCT07474311
• A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular AtrophyCompletedNCT07403214
• A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying TherapiesCompletedNCT07378943
• Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular DystrophyActive, not recruitingNCT07356063Not applicable
• Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).WithdrawnNCT07265232Phase 3
• Gut Peptides and Bone Remodeling in Children With Neuromuscular DisordersTerminatedNCT07254988Not applicable
• HABIT-ILE + FST in Children With SMAActive, not recruitingNCT07223320Not applicable
• Impact of Motor and Oral Motor Function on Quality of Life in Children With SMACompletedNCT06864767
• Efficacy and Usability of the EXPLORER Exoskeleton in Children With Neurodevelopmental Disorders in Natural EnvironmentsActive, not recruitingNCT06765070Not applicable
• Robot-assisted TrainingCompletedNCT06648486Not applicable
• Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in SpainCompletedNCT06632730
• Ultrasonographic Muscle Assessment and Functional Scales in Spinal Muscular AtrophyActive, not recruitingNCT06599606
• Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type ICompletedNCT06576388Early Phase 1
• Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA).CompletedNCT06419322Not applicable
• The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular DisorderCompletedNCT06363357Not applicable
• Digital Assessment of Speech and Fine Motor Control in ALSCompletedNCT06315673
• Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper LimbCompletedNCT06300996Not applicable
• An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular AtrophyActive, not recruitingNCT06288230Phase 1/2
• Two Different Treatment Modalities in Patients With Spinal Muscular AtrophyCompletedNCT06178653Not applicable
• Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United StatesCompletedNCT06178393
• Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular Disease in Their Natural EnvironmentCompletedNCT06167954Not applicable
• Genetic Newborn Screening for Cystinosis and Spinal Muscular AtrophyCompletedNCT06027385Not applicable
• The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular AtrophyCompletedNCT05878418Not applicable
• A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene TherapyActive, not recruitingNCT05861999Phase 4
• Digital Smartwatch Measurements as Potential Biomarkers for Remote Disease Tracking in ALSWithdrawnNCT05830214
• A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular AtrophyCompletedNCT05808764Phase 2
• Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular AtrophyCompletedNCT05794139Phase 2
• Personalized Medicine for SMA: a Translational ProjectStatus unknownNCT05779956
• SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy PatientsStatus unknownNCT05761262
• SMN Circular RNAs as Potential New Targets and Biomarkers for SMAStatus unknownNCT05760209
• Body Weight Support Harness System in Spinal Muscular AtrophyCompletedNCT05715749Not applicable
• Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIVCompletedNCT05712330Not applicable
• Feasibility of a Newborn Screening for Spinal Muscular Atrophy (SMA) in France: DEPISMA Prefigurator Project in Grand-Est and Nouvelle-AquitaineStatus unknownNCT05645250
• Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy (The EchoSpin Study)CompletedNCT05644899
• Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA).Status unknownNCT05632666Not applicable
• Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of ApitegromabActive, not recruitingNCT05626855Phase 3
• Adult Spinal Muscular Atrophy (SMA) China RegistryActive, not recruitingNCT05618379
• Spinal Muscular Atrophy on Wheels, Using Power MobilityStatus unknownNCT05589987Not applicable
• 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 AbeparvovecActive, not recruitingNCT05575011Phase 1
• Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-oldStatus unknownNCT05539456
• Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With NusinersenActive, not recruitingNCT05522361Phase 4
• Myotonometer Intra- and Inter Examiner Reliability on Spinal Muscular AtrophyStatus unknownNCT05521217
• Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular AtrophyCompletedNCT05518773
• Newborn Screening for Spinal Muscular AtrophyCompletedNCT05481164
• Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin AmericaActive, not recruitingNCT05475691
• Exoskeleton Impact on the Quality of Life on Patients With Spinal Muscular AtrophyCompletedNCT05416034Not applicable
• Schulze Muscular Dystrophy Ability Clinical StudyStatus unknownNCT05409079Not applicable
• Quality of Life and Participation of the Adult with Spinal Muscular Atrophy in FranceCompletedNCT05366465
• Virtual Reality for the Mitigation of Anxiety During Intrathecal Administration in Participants With Spinal Muscular AtrophyCompletedNCT05354414Not applicable
• Equine-Assisted Physiotherapy for Spinal Muscular AtrophyCompletedNCT05341453Not applicable
• A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular AtrophyActive, not recruitingNCT05337553Phase 3
• Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical TrialsActive, not recruitingNCT05335876Phase 3
• Procedural Sedation for Pediatric Patients With Spinal Muscular AtrophyCompletedNCT05291962
• A BHI to Increase Hope Level and Stress Level of Parents With a CMCCompletedNCT05269693Not applicable
• Newborn Screening for Spinal Muscular Atrophy (SMA) - a Proof of Principle Study Using Anonymised Blood Spots.CompletedNCT05237544
• Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)CompletedNCT05232929Phase 4
• Investigating NMJ Defects in SMA Following Central and Peripheral SMN RestorationCompletedNCT05219487
• Antisense Oligonucleotide for Spinal Muscular AtrophyStatus unknownNCT05187260
• Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or RisdiplamCompletedNCT05156320Phase 3
• Pediatric Powered Wheelchair Standing Devices: An Exploratory StudyActive, not recruitingNCT05117827Not applicable
• A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular AtrophyActive, not recruitingNCT05115110Phase 2/3
• Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy PracticesStatus unknownNCT05110885
• A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular DisordersCompletedNCT05109637Not applicable
• 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)Active, not recruitingNCT05067790Phase 3
• Pediatric Spinal Muscular Atrophy (SMA) China RegistryActive, not recruitingNCT05042921
• SWITCH II Early Feasibility Study: Implantable BCI to Control a Digital Device for People With ParalysisWithdrawnNCT05041114Not applicable
• COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With ParalysisActive, not recruitingNCT05035823Not applicable
• Musculoskeletal Nociceptive Pain in Participants With Neuromuscular DisordersCompletedNCT04907162
• Holter of Movement in Patients with SMA Undergoing Treatment.Active, not recruitingNCT04888702Not applicable
• Phenotype, Genotype and Biomarkers 2Active, not recruitingNCT04875416
• Prenatal Carrier Screening for Spinal Muscular Atrophy Among Thai Pregnant WomenStatus unknownNCT04859179
• Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative TherapiesActive, not recruitingNCT04833348Not applicable
• NuroSleeve Powered Brace & Stimulation System to Restore Arm FunctionActive, not recruitingNCT04798378Not applicable
• Implementation of FEES in Spinal Muscle AtrophyCompletedNCT04773470Not applicable
• A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)CompletedNCT04729907Phase 3
• Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy ParticipantsCompletedNCT04718181Phase 1

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