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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: SMA - Spinal Muscular Atrophy Clear all
Recruiting Phase 3 Interventional

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)

NCT07221669
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Salanersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
0 Days to 42 Days
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
3
States / cities
Chicago, Illinois • Flower Mound, Texas • Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Completed Phase 2 Interventional Results available

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

NCT02386553
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
0 Weeks to 6 Weeks
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
11
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2025 · Synced Sep 17, 2026, 6:02 AM EDT
Recruiting No phase listed Observational

Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

NCT04174157
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
Prospective observational registry, Zolgensma
Other · Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2038
U.S. locations
50
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Loma Linda, California + 45 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2025 · Synced Sep 17, 2026, 6:02 AM EDT
Active, not recruiting Phase 3 Interventional

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

NCT04042025
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III, SMA
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Not listed
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
18
States / cities
Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 14 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2025 · Synced Sep 17, 2026, 6:02 AM EDT
Recruiting Phase 4 Interventional

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

NCT05861986
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months to 24 Months
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
11
States / cities
Little Rock, Arkansas • Aurora, Colorado • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Completed Phase 2 Interventional Results available

A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)

NCT01839656
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
21 Days to 210 Days
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
3
States / cities
Stanford, California • Orlando, Florida • New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Sep 17, 2026, 6:02 AM EDT
Recruiting Phase 3 Interventional

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 Abeparvovec

NCT07444450
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Salanersen, Sham Procedure
Drug · Procedure
Lead sponsor
Biogen
Industry
Eligibility
0 Days to 7 Months
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2033
U.S. locations
1
States / cities
Flower Mound, Texas
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

NCT00439569
View directory record Official record
Conditions
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
5
States / cities
Stanford, California • St Louis, Missouri • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 7, 2010 · Synced Sep 17, 2026, 6:02 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Prospective Evaluation of Infants With Spinal Muscular Atrophy:

NCT02831296
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 1, 2020 · Synced Sep 17, 2026, 6:02 AM EDT
Not listed Not applicable Interventional

Schulze Muscular Dystrophy Ability Clinical Study

NCT05409079
View directory record Official record
Conditions
Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy, FSHD, Cerebral Palsy, Becker Muscular Dystrophy
Interventions
Abilitech Assist
Device
Lead sponsor
AbiliTech Medical Inc.
Industry
Eligibility
10 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2022 · Synced Sep 17, 2026, 6:02 AM EDT
Completed Phase 2 Interventional

Levetiracetam for Cramps, Spasticity and Neuroprotection in Motor Neuron Disease

NCT00324454
View directory record Official record
Conditions
Motor Neuron Disease, Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy
Interventions
Levetiracetam
Biological
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jun 18, 2013 · Synced Sep 17, 2026, 6:02 AM EDT
Completed Phase 1 Interventional

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)

NCT02052791
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
Not listed
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 15, 2021 · Synced Sep 17, 2026, 6:02 AM EDT
Recruiting No phase listed Observational

A Registry of Cases of Spinal Muscular Atrophy

NCT07805278
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
30 participants
Timeline
2026 – 2033
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 7, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Completed No phase listed Observational

Adults With SMA Treated With Nusinersen

NCT04591678
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Ohio State University
Other
Eligibility
18 Years to 60 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 21, 2021 · Synced Sep 17, 2026, 6:02 AM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

NCT04851873
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Up to 17 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Boston, Massachusetts • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Sep 17, 2026, 6:02 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

TRIAL READY (Clinical Trial Readiness)

NCT03912987
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, ALS-Frontotemporal Dementia, Primary Lateral Sclerosis, Progressive Muscular Atrophy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
18 Years and older
Enrollment
610 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2031
U.S. locations
3
States / cities
Miami, Florida • Kansas City, Kansas • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Completed Not applicable Interventional Results available

Progressive Strength Training in Spinal Muscular Atrophy

NCT01233817
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Progressive strength training
Other
Lead sponsor
University of Utah
Other
Eligibility
5 Years to 21 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Jun 13, 2017 · Synced Sep 17, 2026, 6:02 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy

NCT00528268
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Sodium phenylbutyrate
Drug
Lead sponsor
University of Utah
Other
Eligibility
1 Day to 6 Months
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Feb 27, 2025 · Synced Sep 17, 2026, 6:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Digital Assessment of Speech and Fine Motor Control in ALS

NCT06315673
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy
Interventions
Digital Speech and Handwriting Assessment
Behavioral
Lead sponsor
Milton S. Hershey Medical Center
Other
Eligibility
18 Years and older
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2026
U.S. locations
1
States / cities
Hershey, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 28, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

NCT02327845
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Progressive Muscular Atrophy, Multisystem Proteinopathy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
Not listed
Enrollment
708 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2031
U.S. locations
13
States / cities
Palo Alto, California • San Diego, California • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Terminated Phase 1 Interventional Results available

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

NCT03381729
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
6 Months to 60 Months
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
11
States / cities
Los Angeles, California • Stanford, California • Orlando, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Active, not recruiting Phase 3 Interventional

A 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)

NCT05067790
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
15 Years to 50 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Atlanta, Georgia + 8 more
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Recruiting No phase listed Observational

Gait and Bone Health in SMA

NCT07400198
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Ambulatory Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Jacqueline Montes
Other
Eligibility
8 Years to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Sep 17, 2026, 6:02 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)

NCT01166022
View directory record Official record
Conditions
Spinal Muscular Atrophy, Neuromuscular Disease
Interventions
Exercise
Other
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 50 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2014
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 11, 2016 · Synced Sep 17, 2026, 6:02 AM EDT