Muscular Atrophy, Spinal clinical trials
The registry lists 282 studies for Muscular Atrophy, Spinal across more than 10 countries. 73 are recruiting or about to start. 204 of them are mainly about Muscular Atrophy, Spinal (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12. Counting its 2 narrower conditions too, such as Spinal Muscular Atrophies of Childhood: 361 studies, 94 open.
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Status
Who runs them
Biogen (27) · Hoffmann-La Roche (16) · Novartis Pharmaceuticals (14) · Columbia University (9) · Fondazione Policlinico Universitario Agostino Gemelli IRCCS (9) · University of Utah (6) · Institut de Myologie, France (5) · National Taiwan University Hospital (5)
What is being tested here
Drugs under test in Muscular Atrophy, Spinal studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
The 10 nearest, phase 3 first
- Nov 2026Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab · Phase 3 · Scholar Rock, Inc.
- Dec 2026Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III · Phase 3 · GeneCradle Inc
- Sep 2027A 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) · Phase 3 · Biogen
- Nov 2026Spinal Muscular Atrophy and School Transitions · Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust
- Nov 2026Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy · Aga Khan University
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Dec 2026Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMA · Istituto Giannina Gaslini
- Dec 2026Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases · IRCCS Eugenio Medea
- Dec 2026CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain · Fundació Sant Joan de Déu
- Dec 2026Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients · GeneCradle Inc
Who these studies take
Of the 58 Muscular Atrophy, Spinal studies recruiting now, 28 (48%) set no upper age limit and 27 (47%) stop at 75 or younger. 46 (79%) take children under 18. 1 (2%) are for women only and 1 (2%) for men only; 11 (19%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 29 (50%) | 29 (50%) |
| 18 | 39 (67%) | 39 (67%) |
| 30 | 37 (64%) | 37 (64%) |
| 40 | 37 (64%) | 37 (64%) |
| 50 | 37 (64%) | 37 (64%) |
| 65 | 34 (59%) | 34 (59%) |
| 75 | 32 (55%) | 32 (55%) |
| 85 | 30 (52%) | 30 (52%) |
Across all 282 studies, recruiting or not: 57% stop at 75 or younger and 52% at 65; 79% take children; 1% are for women only and 1% for men only; 21% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
How long Muscular Atrophy, Spinal studies take
Of 18 phase 3 interventional studies under Muscular Atrophy, Spinal, half reached primary completion within 33 months of starting, counting those still running.
| Start to primary completion | Every sponsor | Industry | Non-industry |
|---|---|---|---|
| Phase 2 | 34 mo25–42 · 15 studies | — | — |
| Phase 3 | 33 mo24–63 · 18 studies | — | — |
| Interventional, no phase | 27 mo17–51 · 57 studies | — | 28 mo19–52 · 48 studies |
| Observational | 88 mo36–… · 58 studies | — | — |
Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out
What changed recently
From the registry's own updates since Oct 14, 2026.
Started recruiting2
Results posted1
Stopped1
- Home Monitoring of Adult Patients With SMA: a Pilot Multicenter Validation StudyWithdrawnNov 4, 2026NCT05839145
Newly registered5
- SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)Oct 29, 2026NCT07903376
- Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System DisordersOct 28, 2026NCT07900672
- SMAdult - Monitoring of Movement on Spinal Muscular AtrophyOct 26, 2026NCT07894185
- Service Evaluation for Newborn Screening for Spinal Muscular AtrophyOct 22, 2026NCT07888868
- Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMAOct 20, 2026NCT07883655
Studies
Recruiting first, then most recently updated. Showing 50 of 282. Browse all 282 → or search them.
- 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 BabiesRecruitingNCT057897582 countries
- A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi ArabiaNot yet recruitingNCT07737756
- Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular AtrophyRecruitingNCT05866419Not applicable5 countries
- A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)RecruitingNCT075317191 country
- Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in Volunteers With Neuromuscular Diseases or Asymptomatic VolunteersRecruitingNCT07321977Not applicable1 country
- 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)RecruitingNCT075430031 country
- 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 41 country
- SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)Not yet recruitingNCT07903376Early Phase 11 country
- Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System DisordersRecruitingNCT079006721 country
- SMAdult - Monitoring of Movement on Spinal Muscular AtrophyRecruitingNCT07894185Not applicable1 country
- 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 37 countries
- Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMARecruitingNCT07822711Not applicable1 country
- A Registry of Cases of Spinal Muscular AtrophyRecruitingNCT078052781 country
- HABIT-ILE + FST in Children With SMA: Preliminary EffectivenessRecruitingNCT07488338Not applicable1 country
- Service Evaluation for Newborn Screening for Spinal Muscular AtrophyRecruitingNCT078888681 country
- A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy PatientsRecruitingNCT077678901 country
- Advanced Multi-omics and -System Approaches to Identify Novel Biomarkers for SMARecruitingNCT078836551 country
- 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 31 country
- Characterizing Perceived Physical Fatigability in Nusinersen-treated SMARecruitingNCT069558971 country
- Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)RecruitingNCT07521930Not applicable1 country
- 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 24 countries
- A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular AtrophyRecruitingNCT07047144Phase 27 countries
- 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 32 countries
- An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058)Not yet recruitingNCT07846696Phase 31 country
- 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 applicable1 country
- 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 17 countries
- UK SMA Patient RegistryRecruitingNCT042925741 country
- Spinal Muscular Atrophy and School TransitionsNot yet recruitingNCT077205181 country
- Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed TherapiesRecruitingNCT065324741 country
- Clinical Procedures to Support Research in ALSRecruitingNCT034892781 country
- ALS/MND Natural History Study Data RepositoryRecruitingNCT059660383 countries
- Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular AtrophyRecruitingNCT06562283Not applicable1 country
- Adrenal& Bone Complications in Paediatric Patients Living With Spinal Muscular Atrophy (SMA)Not yet recruitingNCT075309261 country
- Invasive Home Ventilation in DenmarkRecruitingNCT074671871 country
- Comprehensive Analysis Platform To Understand, Remedy and Eliminate ALSRecruitingNCT052040171 country
- Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular DisordersRecruitingNCT068394691 country
- Gait and Bone Health in SMARecruitingNCT074001981 country
- Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring TherapyRecruitingNCT06321965Not applicable1 country
- Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular DiseasesRecruitingNCT066668161 country
- Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health SystemRecruitingNCT072234891 country
- Neuroproprioceptive Equine-Assisted Physiotherapy for Spinal Muscular AtrophyNot yet recruitingNCT07336602Not applicable1 country
- Long Read Analysis in Spinal Muscular Atrophy - LOREASIRecruitingNCT07332702Not applicable1 country
- Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn ScreeningRecruitingNCT07286565Not applicable1 country
- CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in SpainRecruitingNCT072315491 country
- Psychological Evaluation of the Parental Experience of Newborn Screening for Infantile Spinal Muscular Atrophy in the Grand Est and Nouvelle-Aquitaine RegionsNot yet recruitingNCT07208903
- Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular DisordersEnrolling by invitationNCT066056121 country
- Safety, Usability, and Effectiveness of a Gait Exoskeleton for Children and Adolescents With Neurodevelopmental Disorders.Not yet recruitingNCT07159360Not applicable2 countries
- The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.Enrolling by invitationNCT07072676Not applicable1 country
- A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With Spinal Muscular AtrophyRecruitingNCT06396325Not applicable1 country
- Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 PatientsRecruitingNCT05824169Phase 1/21 country