Also listed under Motor Neuron Disease
ClinicalTrials.gov term: Muscular Atrophy, Spinal
Spinal Muscular Atrophy clinical trials
Data updated daily from ClinicalTrials.gov, last refresh 2026-11-06
What changed in the last 30 days
17 trials changed in the last 30 days: 0 stopped, 7 with notable changes, 10 with minor changes only.
- Minor: MANATEE · RO7204239 in Combination With Risdiplam (RO7034067) · Spinal Muscular Atrophy Trial completed
- Minor: Myokinesis · Portable Digital Device for Quantified Analysis of Markerless Walking · Neuromuscular Diseases or Asymptomatic Volunteers Primary completion pushed from Jul 6, 2026 to Dec 31, 2026
- Minor: SMOB · Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients Trial status changed: Unknown → Completed
- Minor: REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM) Trial sites expanded: 1 → 6 locations
- Notable: Spinal Cord Stimulation · Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb Primary endpoint(s) modified
- Notable: Different Stiffness Settings During Robotic Isokinetic Training · SMA Recruitment opened
- Minor: STELLAR-1 · Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA) Trial sites expanded: 11 → 14 locations
- Notable: Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients Recruitment opened
- Minor: HINALEA 2 · Risdiplam Administered · Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy Enrollment closed, study ongoing
- Minor: WeSMA · Follow-up Study of Risdiplam · Spinal Muscular Atrophy (SMA) Trial completed
- Minor: Registry of Cases of Spinal Muscular Atrophy Start date pushed from Oct 2026 to Nov 2026
- Minor: DANNIgene · Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders Study completion pushed from May 2027 to Jun 2028
- Notable: Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA Primary completion pushed from Dec 7, 2026 to Sep 6, 2027
- Notable: SMA-REAL · Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia Start date pushed from Aug 25, 2026 to Nov 1, 2026
- Notable: SEE-NMD · Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers · Neuro Muscular Dystrophy Primary completion pushed from Mar 23, 2026 to Nov 20, 2026
- Notable: INTENT · Interfacing With NeuroTechnology to Expand Neural Throughput Start date pushed from Sep 2026 to Jan 2027
- Minor: Sparkle · Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy Trial sites expanded: 23 → 28 locations
- Stopped: terminated, suspended or withdrawn
- Notable: the plan changed
- Minor: routine updates
New sponsor entrants, last 90 days sponsors with their first trial in this area
| First posted | Trial | Sponsor | Phase |
|---|---|---|---|
| 2026-10-29 | NCT07903376 SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE) | Exir Gene Pharmed new | Early Phase 1 |
| 2026-10-28 | NCT07900672 Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other… | Ambulanzpartner Soziotechnologie APST GmbH new | |
| 2026-10-20 | NCT07883655 Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA | Istituto Giannina Gaslini new | |
| 2026-09-24 | NCT07838779 An OLIP Study (Open Label Individual Patient) of Nusinersen in a Participant With Spinal… | London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's new |
Active trials by phase
| Early Phase 1 | 2 | |
| Phase 1 | 2 | |
| Phase 1/2 | 7 | |
| Phase 2 | 4 | |
| Phase 2/3 | 1 | |
| Phase 3 | 10 | |
| Phase 4 | 3 |
Not charted: 82 active trials with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).
Top sponsors by active trials in this area
Biogen (10) · Novartis Pharmaceuticals (5) · Assistance Publique - Hôpitaux de Paris (4) · GeneCradle Inc (4) · Hoffmann-La Roche (4) · University of Miami (4) · University of Oxford (3) · Centre Hospitalier Universitaire de Liege (2) · Columbia University (2) · Fondazione Policlinico Universitario Agostino Gemelli IRCCS (2)
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