Disease landscape

Spinal Muscular Atrophy clinical trials

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94 active trials 51 recruiting 11 Phase 3 60 sponsors 22 changes in 30 days

Data updated daily from ClinicalTrials.gov, last refresh 2026-09-16

What changed in the last 30 days

2026-08-25
Primary completion pushed: 2027-06-25 → 2027-09-30
Enrollment increased: 58 → 78 participants
Completion pushed: 2027-06-25 → 2027-09-30
Trial arms changed: 1 → 2
NCT06555419 A Study to Find Out How Nusinersen is Processed in the Body When Given Through the… · Biogen · Phase 1
2026-09-12
Primary completion pushed: 2026-12 → 2027-12
NCT06772402 GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy · Genecombio Ltd.
2026-08-21
Primary completion pushed: 2025-05-31 → 2040-01-01
Trial status changed: Unknown → Recruiting
Completion pushed: 2025-05-31 → 2040-01-01
NCT04292574 UK SMA Patient Registry · Newcastle University
2026-08-27
Recruitment opened
NCT07554924 A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal… · Lanyue Biotech (Hangzhou) Co., Ltd. · Phase 1/2
2026-09-16
Enrollment closed, study ongoing
NCT07578454 VRehab-SMA Phase 1.2 · University of Oxford
2026-09-16
Trial completed
Study completion confirmed: August 2026 (last estimated December 2026)
NCT06419322 Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for… · University of Oxford
2026-09-09
Recruitment opened
NCT07805278 A Registry of Cases of Spinal Muscular Atrophy · St. Jude Children's Research Hospital
2026-09-04
Trial completed
Final enrollment: 8 participants (planned 30)
NCT06363357 The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements… · Seoul National University Hospital
2026-09-10
Trial sites reduced: 35 → 30 locations
Final enrollment: 97 participants (planned 259)
NCT05115110 A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam… · Hoffmann-La Roche · Phase 2/3
2026-08-19
Trial sites expanded: 7 → 11 locations
NCT07221669 A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When… · Biogen · Phase 3
2026-09-10
Primary completion moved earlier: 2026-12-31 → 2026-10-02
Completion moved earlier: 2026-12-31 → 2026-10-02
NCT05232929 Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) · Genentech, Inc. · Phase 4
2026-09-09
Completion pushed: 2026-09 → 2028-09
NCT05035823 COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People… · Synchron Medical, Inc.
2026-09-06
Trial status changed: Active, Not Recruiting → Unknown
NCT04690998 Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV… · Assistance Publique Hopitaux De Marseille

New sponsor entrants, last 90 days sponsors with their first trial in this area

First postedTrialSponsorPhase
2026-07-22 NCT07720518 Spinal Muscular Atrophy and School Transitions Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust new

Active trials by phase

Early Phase 1
1
Phase 1
2
Phase 1/2
8
Phase 2
3
Phase 2/3
1
Phase 3
10
Phase 4
4
Not charted: 65 active trials with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).

Active trials top 25 of 94

NCT IDTitleSponsorStatusUpdated
Phase 4
NCT05861999 A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric… Hoffmann-La Roche Recruiting 2026-09-10
NCT05861986 A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early… Hoffmann-La Roche Recruiting 2026-09-03
NCT05232929 Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) Genentech, Inc. Active, Not Recruiting 2026-09-09
NCT07448610 ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants… Novartis Pharmaceuticals Not Yet Recruiting 2026-05-29
Phase 3
NCT07221669 A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When… Biogen Recruiting 2026-08-18
NCT07444450 A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to… Biogen Recruiting 2026-08-11
NCT07444476 A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in… Biogen Recruiting 2026-04-21
NCT07265232 Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal… Lantu Biopharma Recruiting 2025-12-04
NCT06971094 Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the… GeneCradle Inc Recruiting 2025-06-05
NCT05626855 Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous… Scholar Rock, Inc. Active, Not Recruiting 2026-09-01
NCT05335876 Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in… Novartis Pharmaceuticals Active, Not Recruiting 2026-07-09
NCT05337553 A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With… Biohaven Pharmaceuticals, Inc. Active, Not Recruiting 2026-06-01
NCT05067790 A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as… Biogen Active, Not Recruiting 2026-05-22
NCT04042025 Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi Novartis Gene Therapies Active, Not Recruiting 2025-04-25
Phase 2/3
NCT05115110 A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam… Hoffmann-La Roche Active, Not Recruiting 2026-09-09
Phase 2
NCT07047144 A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and… Scholar Rock, Inc. Recruiting 2026-09-14
NCT07287982 A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and… argenx Recruiting 2026-09-14
NCT03779334 A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal… Hoffmann-La Roche Active, Not Recruiting 2026-07-09
Phase 1/2
NCT07554924 A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal… Lanyue Biotech (Hangzhou) Co., Ltd. Recruiting 2026-08-26
NCT07070999 Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular… Gemma Biotherapeutics Recruiting 2026-06-29
NCT05824169 Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… GeneCradle Inc Recruiting 2025-07-03
NCT06421831 Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… GeneCradle Inc Recruiting 2025-07-03
NCT05747261 Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN… Biocad Recruiting 2024-02-22
NCT06288230 An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal… Lantu Biopharma Active, Not Recruiting 2026-06-15
NCT05901987 Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… GeneCradle Inc Active, Not Recruiting 2025-07-03
See all 94 active Spinal Muscular Atrophy trials with filters →

Top sponsors by active trials in this area

Biogen (10) · Hoffmann-La Roche (5) · Novartis Pharmaceuticals (5) · Assistance Publique - Hôpitaux de Paris (4) · GeneCradle Inc (4) · Guangzhou Jiayin Biotech Ltd (3) · Columbia University (2) · Fondazione Policlinico Universitario Agostino Gemelli IRCCS (2) · Lantu Biopharma (2) · LMU Klinikum (2)

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