Clinical Trials
EXTERNAL DATASMA clinical trials aggregated from ClinicalTrials.gov via the v2 API with automated daily refresh. Covers all interventional and observational studies related to spinal muscular atrophy — from early Phase 1 safety trials through Phase 3 efficacy studies and post-marketing surveillance. Each trial entry includes NCT identifier, phase, enrollment, status, intervention type, primary/secondary outcome measures, and where available, published results with adverse events and participant flow data. Use the filters to explore by phase, status, intervention type, or keyword.
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| NCT ID | Title | Phase | Status | Sponsor | N | |
|---|---|---|---|---|---|---|
| NCT07444450 | A Phase 3, Randomized, Double-Blinded Study to Evaluate the Safety and Efficacy of Salanersen (BIIB115) After Onasemnogene Abeparvovec Treatment in Infants With Genetically Diagnosed Spinal Muscular Atrophy | Phase 3 | not yet recruiting | Biogen | 42 | |
| NCT07448610 | Onasemnogene Abeparvovec: ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM) | Phase 4 | not yet recruiting | Novartis Pharmaceuticals | 36 | |
| NCT07325149 | Exoskeletal-assisted Walking Combined With Transcutaneous Spinal Cord Stimulation: Effect on Imaging and Serum Biomarkers of Skeletal Muscle Mass and Bone Strength. | NA | not yet recruiting | Bronx VA Medical Center | 24 | |
| NCT05470478 | Enhancement and Optimization of a Mobile iBCI for Veterans With Paralysis | NA | not yet recruiting | VA Office of Research and Development | 2 | |
| NCT07320378 | Clinical Study on the Safety and Efficacy of Tendon Stem Progenitor Cells Therapy for Rotator Cuff Tears | EARLY_Phase 1 | not yet recruiting | Second Affiliated Hospital, School of Medicine, Zhejiang University | 107 | |
| NCT07336602 | Immediate and Sustained Effects of Intensive Neuroproprioceptive Equine-Assisted Physiotherapy in Children With Spinal Muscular Atrophy: A Randomized Crossover Trial | NA | not yet recruiting | Charles University, Czech Republic | 20 | |
| NCT07444476 | An Open-Label, Phase 3 Study to Evaluate the Efficacy and Safety of Salanersen (BIIB115) in Participants Aged 15-60 Years With Spinal Muscular Atrophy Who Are Either Treatment-Naïve or Have Previously Been Treated With Risdiplam | Phase 3 | not yet recruiting | Biogen | 90 | |
| NCT07467187 | The Evolution of Invasive Home Mechanical Ventilation in Denmark | — | not yet recruiting | Rigshospitalet, Denmark | 450 | |
| NCT06998134 | Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults: Exploratory Observational Analysis of Novel Robotic Control Strategies | — | recruiting | National Institutes of Health Clinical Center (CC) | 23 | |
| NCT07488338 | Feasibility and Preliminary Effectiveness of HABIT-ILE + Functional Strength Training in Children With Spinal Muscular Atrophy (SMA): a Prospective Single-group Intervention Trial. | NA | recruiting | Teachers College, Columbia University | 20 | |
| NCT07478172 | Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults withNeuromuscular Disease | NA | recruiting | University of Missouri-Columbia | 50 | |
| NCT07356063 | Effect of a Multicomponent Support Group on Self-Efficacy of Primary Caregivers of Children With Spinal Muscular Atrophy or Duchenne Muscular Dystrophy Enrolled in Treat-NMD Registry of Pakistan | NA | not yet recruiting | Aga Khan University | 30 | |
| NCT07400198 | Identification of Gait-related Digital Biomarkers of Bone Health in Spinal Muscular Atrophy | — | recruiting | Jacqueline Montes | 22 | |
| NCT07221669 | An Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular Atrophy | Phase 3 | recruiting | Biogen | 30 | |
| NCT07190300 | TulmiSTAR-02: A Two-part Phase I Dose Escalation Study of Tulmimetostat (DZR123) in Combination With Darolutamide or Abiraterone Followed by Open-label, Randomized, Phase II Dose Expansion Study to Assess the Safety and Efficacy of Tulmimetostat in Combination With Darolutamide Versus Darolutamide Alone in Patients With Metastatic Hormone-sensitive Prostate Cancer | Phase 1, Phase 2 | recruiting | Novartis Pharmaceuticals | 155 | |
| NCT07070999 | A Phase 1-2, Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of GB221 Delivered Into the Cisterna Magna of Pediatric Participants From 2 Weeks to Younger Than 12 Months of Age With Spinal Muscular Atrophy Type 1 | Phase 1, Phase 2 | recruiting | Gemma Biotherapeutics | 22 | |
| NCT07336446 | A Phase I/II, Modular, Open-Label, Multi-Centre Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of AZD9750 as Monotherapy and in Combination With Other Anticancer Agents in Participants With Metastatic Prostate Cancer (ANDROMEDA) | Phase 1, Phase 2 | not yet recruiting | AstraZeneca | 300 | |
| NCT07321977 | Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers | NA | recruiting | Institut de Myologie, France | 30 | |
| NCT07327723 | The Effects of Repetitive Peripheral Magnetic Stimulation on Clinical and Electrophysiological Parameters in Patients With Carpal Tunnel Syndrome: A Double-Blind, Randomized, Sham-Controlled Trial | NA | not yet recruiting | Ankara University | 40 | |
| NCT07287982 | A Phase 2 Double-Blinded, Randomized, Placebo-Controlled 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 Atrophy | Phase 2 | recruiting | argenx | 60 | |
| NCT07239427 | Use of Transcranial Direct Current Stimulation (TDCS) in an Active Pain Coping Program for Patients With Fibromyalgia: A Randomized Control Trial. | NA | recruiting | University of Valladolid | 68 | |
| NCT07159555 | Neurofilaments Levels in Premature Newborns | — | recruiting | Centre Hospitalier Universitaire de Liege | 15 | |
| NCT07286565 | Active-NBS Liege - Monitoring the Motor Development of Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified Through Newborn Screening | NA | recruiting | Centre Hospitalier Universitaire de Liege | 100 | |
| NCT07408570 | FREQUENCY- DEPENDENT MODULATION OF SOLEUS REFLEX RESPONSES DURING WHOLE BODY- VIBRATION IMPLICATIONS FOR PORPELASTIC BOBE MYOREGULATION UNDER GRAVITATIONAL LOADING | NA | enrolling by invitation | Istanbul Physical Medicine Rehabilitation Training and Research Hospital | 27 | |
| NCT03940911 | Fatigue and Skeletal Muscle Impact in Severe Axial Spondyloarthritis | NA | recruiting | University Hospital, Strasbourg, France | 122 |
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