15 recruiting, 5 active not recruiting, 4 completed, 2 enrolling by invitation. 15 Not applicable, 4 Phase 2, 3 Phase 3, 1 Early Phase 1, 1 Phase 1.
Follow Spinal Muscular Atrophy (SMA): new trials by email Literature Review of Spinal Muscular Atrophy (SMA) trials Research Report on Spinal Muscular Atrophy (SMA)
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMA Portable Isokinetic Knee Training Robot With an Individually Optimized Compliant Setting, Portable Isokinetic Knee Training Robot With a Rigid Setting - Spinal Muscular Atrophy (SMA) | Not applicable | Recruiting | Peking University Third Hospital | 13 | 2026-10-22 | 2026-10-23 |
| Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy Multicomponent Intervention - Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA) | Not applicable | Active not recruiting | Aga Khan University | 34 | 2026-08-15 | 2026-10-09 |
| Service Evaluation for Newborn Screening for Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA) | Recruiting | University of Oxford | 750000 | 2026-08-01 | 2026-10-22 | |
| VRehab-SMA Phase 1.2 VRehab SMA device - Spinal Muscular Atrophy (SMA) | Not applicable | Active not recruiting | University of Oxford | 12 | 2026-05-27 | 2026-09-15 |
| 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) Spinal Muscular Atrophy (SMA) | Recruiting | University of Oxford | 90 | 2026-05-01 | 2026-05-11 | |
| Neuroproprioceptive Equine-Assisted Physiotherapy for Spinal Muscular Atrophy Equine-Assisted Physiotherapy based on Neuro-proprioceptive "Facilitation and Inhibition", Standard Individual Outpatient Physiotherapy Based on Neuro-proprioceptive Facilitation and Inhibition, Therapeutic grooming - Spinal Muscular Atrophy (SMA) | Not applicable | Not yet recruiting | Charles University, Czech Republic | 20 | 2026-04-15 | 2026-01-13 |
| HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness Combined Hand-Arm Bimanual Intensive Therapy Including Lower Extremities (HABIT-ILE) + functional strength training (FST) - Spinal Muscular Atrophy (SMA) | Not applicable | Recruiting | Teachers College, Columbia University | 20 | 2026-03-15 | 2026-10-22 |
| Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic Volunteers Gait analysis via video capture - Spinal Muscular Atrophy (SMA), Charcot-Marie-Tooth | Not applicable | Recruiting | Institut de Myologie, France | 30 | 2026-03-03 | 2026-05-19 |
| 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 Atrophy ARGX-119 IV, Placebo IV - Spinal Muscular Atrophy (SMA) | Phase 2 | Recruiting | argenx | 60 | 2025-12-19 | 2026-10-08 |
| Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening MAIJU, Syde, Questionnaires - Spinal Muscular Atrophy (SMA), Duchenne Muscular Dystrophy (DMD) | Not applicable | Recruiting | Centre Hospitalier Universitaire de Liege | 100 | 2025-12-01 | 2025-12-16 |
| Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine Regions focus group discussions, Interview with the psychologist - Spinal Muscular Atrophy (SMA), Spinal Muscular Atrophy Type I | Not yet recruiting | University Hospital, Strasbourg, France | 36 | 2025-10-20 | 2025-10-06 | |
| Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC). vesemnogene lantuparvovec - Spinal Muscular Atrophy (SMA) | Phase 3 | Withdrawn | Lantu Biopharma | 2025-10-15 | 2026-09-25 | |
| Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System Telephone Interview - Spinal Muscular Atrophy (SMA) | Recruiting | Medstar Health Research Institute | 200 | 2025-10-10 | 2026-01-16 | |
| The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. Assistive gait devices combined with physiotherapy - Inclusion Body Myositis, Myotonic Dystrophy 1 | Not applicable | Enrolling by invitation | LMU Klinikum | 30 | 2025-09-01 | 2025-07-18 |
| Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic Stimulation in Spinal Muscular Atrophy High-frequency repetitive transcranial magnetic stimulation, lumbar puncture - Spinal Muscular Atrophy (SMA) | Not applicable | Recruiting | Charitable Foundation Children with Spinal Muscular Atrophy | 20 | 2025-05-19 | 2025-06-18 |
| Long Read Analysis in Spinal Muscular Atrophy - LOREASI blood sample - Spinal Muscular Atrophy (SMA) | Not applicable | Recruiting | University Hospital, Rouen | 27 | 2025-05-15 | 2026-01-12 |
| Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy EXG001-307 injection - Spinal Muscular Atrophy (SMA) | Early Phase 1 | Enrolling by invitation | Guangzhou Jiayin Biotech Ltd | 4 | 2025-03-10 | 2025-03-21 |
| Robot-assisted Training With the assistance of wearable isokinetic training robot （ 7 children with SMA type II） - Spinal Muscular Atrophy (SMA) | Not applicable | Completed | Peking University Third Hospital | 13 | 2025-02-25 | 2026-05-22 |
| Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA Spinal Muscular Atrophy (SMA) | Recruiting | Istituto Giannina Gaslini | 200 | 2025-02-05 | 2026-10-20 | |
| Ultrasonographic Muscle Assessment and Functional Scales in Spinal Muscular Atrophy Ultrasonographic Assessment - Spinal Muscular Atrophy (SMA), Neuromuscular Disease | Unknown | Istanbul University - Cerrahpasa | 34 | 2024-05-01 | 2024-09-24 |
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Muscular Atrophy, Spinal (33) · Muscular Dystrophy, Duchenne (4) · Muscular Dystrophies (3) · Neuromuscular Diseases (3) · Charcot-Marie-Tooth Disease (2) · Muscular Diseases (2) · Muscular Dystrophy, Facioscapulohumeral (2) · Myotonic Dystrophy (2) · Spinal Muscular Atrophy, Type IV (2) · Amyotrophic Lateral Sclerosis (1) · Bulbo-Spinal Atrophy, X-Linked (1) · Cerebral Palsy (1) · Disease (1) · Disease Progression (1) · Friedreich Ataxia (1)
Central Nervous System Diseases (33) · Motor Neuron Disease (33) · Nervous System Diseases (33) · Neurodegenerative Diseases (33) · Spinal Cord Diseases (33) · Neuromuscular Diseases (30) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Genetic Diseases, Inborn (9) · Musculoskeletal Diseases (8) · Muscular Disorders, Atrophic (7) · Muscular Diseases (6) · Genetic Diseases, X-Linked (5) · Heredodegenerative Disorders, Nervous System (5) · Muscular Dystrophies (4) · Pathological Conditions, Signs and Symptoms (4)
Blood Specimen Collection (2) · Interviews as Topic (2) · Risdiplam (2) · Zolgensma (2) · Celecoxib (1) · Physical Therapy Modalities (1) · Spinal Puncture (1) · Standard of Care (1) · Surveys and Questionnaires (1) · nusinersen (1)
University of Oxford (3) · Novartis Pharmaceuticals (2) · Peking University Third Hospital (2) · Aga Khan University (1) · Biocad (1) · Biogen (1) · Centre Hospitalier Universitaire de Liege (1) · Charitable Foundation Children with Spinal Muscular Atrophy (1) · Charles University, Czech Republic (1) · Guangzhou Jiayin Biotech Ltd (1) · Hoffmann-La Roche (1) · Hugh McMillan (1) · IRCCS Eugenio Medea (1) · Institut de Myologie, France (1) · Istanbul University - Cerrahpasa (1)
United States (8) · United Kingdom (7) · China (5) · Canada (4) · France (4) · Italy (4) · Australia (3) · Belgium (3) · Japan (3) · Denmark (2) · Netherlands (2) · Poland (2) · Portugal (2) · Russia (2) · South Korea (2)
Andriy V Shatillo, MD, PhD (1) · Arina V Zinkina-Orikhan, PhD (1) · Hugh McMillan, MD (1) · Katerina Marikova, master (1) · Laurent Servais, MD, PhD (1) · Mette Marie Rosenkilde (1) · Nashit Irfan Aziz, MBBS, MSc (1) · Olena V Pisotska, MD, PhD (1) · Prof Chiara Marini-Bettolo, MD, PhD (1) · Sara Khan, MBBS, MD (1) · Stephen J Kolb, MD PhD (1) · Tamara Dangouloff, PhD (1)
PMID 18506712, PMID 20697104, PMID 24675128, PMID 26432577, PMID 26515624, PMID 27769560, PMID 30045959, PMID 30048507, PMID 30430964, PMID 31478199, PMID 32493524, PMID 3277050, PMID 33456041, PMID 33565742, PMID 33741230, PMID 34980538, PMID 35671520, PMID 35927425, PMID 37037685, PMID 37067602, PMID 38671712, PMID 39210748, PMID 41217888, PMID 41678415, PMID 41712782, PMID 41830075, PMID 42265796, PMID 42413222
2025 (2), 2023 (1), U01NS079163 (1)