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Showing 1–24 of 158 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Muscular Atrophy, Progressive Clear all
Completed Not applicable Interventional

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

NCT02428673
View directory record Official record
Conditions
Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced Jun 26, 2026, 4:26 AM EDT
Terminated Phase 2 Interventional Results available

A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

NCT02462759
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen, Sham Procedure
Drug · Procedure
Lead sponsor
Biogen
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
6
States / cities
Los Angeles, California • Hartford, Connecticut • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Jun 26, 2026, 4:26 AM EDT
Recruiting Not applicable Interventional

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

NCT07478172
View directory record Official record
Conditions
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions
Whole-body Electrical Muscle Stimulation Exercise
Device
Lead sponsor
University of Missouri-Columbia
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
1
States / cities
Columbia, Missouri
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Jun 26, 2026, 4:26 AM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

NCT03421977
View directory record Official record
Conditions
Spinal Muscular Atrophy 1
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 24, 2025 · Synced Jun 26, 2026, 4:26 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 Jun 26, 2026, 4:26 AM EDT
Recruiting Phase 2 Interventional

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

NCT07047144
View directory record Official record
Conditions
Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations, Anti-myostatin
Interventions
Apitegromab, Nusinersen, Risdiplam
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
Up to 2 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
14
States / cities
Phoenix, Arizona • Orange, California • Palo Alto, California + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Jun 26, 2026, 4:26 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

NCT05115110
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
RO7204239, Placebo, Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years to 25 Years
Enrollment
259 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
4
States / cities
Orlando, Florida • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 10, 2026 · Synced Jun 26, 2026, 4:26 AM EDT
Completed Not applicable Interventional

Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy

NCT02341053
View directory record Official record
Conditions
Spinal Muscular Dystrophy, Neuromuscular Disability
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Jun 26, 2026, 4:26 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)

NCT04729907
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years and older
Enrollment
115 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
7
States / cities
Sacramento, California • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Jun 26, 2026, 4:26 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients

NCT01645787
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
4-aminopyridine, Placebo
Drug
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 50 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 2, 2024 · Synced Jun 26, 2026, 4:26 AM EDT
Completed Phase 1Phase 2 Interventional

Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy

NCT01369901
View directory record Official record
Conditions
Motor Neuron Disease
Interventions
A functional exercise program, A stretching exercise program
Behavioral
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 90 Years · Male only
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Jun 26, 2026, 4:26 AM EDT
Completed Phase 4 Interventional

A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)

NCT04488133
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Months to 36 Months
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
9
States / cities
Little Rock, Arkansas • Palo Alto, California • Aurora, Colorado + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2025 · Synced Jun 26, 2026, 4:26 AM EDT
Completed Phase 2 Interventional Results available

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

NCT02908685
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Placebo, Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years to 25 Years
Enrollment
231 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
2
States / cities
Palo Alto, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 23, 2024 · Synced Jun 26, 2026, 4:26 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 Jun 26, 2026, 4:26 AM EDT
Completed Phase 1 Interventional

Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

NCT00374075
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Valproic Acid
Drug
Lead sponsor
University of Utah
Other
Eligibility
2 Years and older
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 9, 2023 · Synced Jun 26, 2026, 4:26 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

NCT00439218
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Months to 48 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
5
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 8, 2010 · Synced Jun 26, 2026, 4:26 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 Jun 26, 2026, 4:26 AM EDT
Approved For Marketing No phase listed Expanded access

An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

NCT04256265
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
2 Months and older
U.S. locations
29
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 25 more
Source: ClinicalTrials.gov public record
Updated Oct 4, 2020 · Synced Jun 26, 2026, 4:26 AM EDT
Terminated Phase 2 Interventional Results available

Genomic Guided Therapy With Dasatinib or Nilutamide in Metastatic Castration-Resistant Prostate Cancer

NCT00918385
View directory record Official record
Conditions
Prostate Cancer
Interventions
Nilutamide, Dasatinib
Drug
Lead sponsor
Duke University
Other
Eligibility
18 Years and older · Male only
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2013
U.S. locations
3
States / cities
Durham, North Carolina • Portland, Oregon • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Oct 28, 2014 · Synced Jun 26, 2026, 4:26 AM EDT
Active, not recruiting Phase 4 Interventional

Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

NCT05232929
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
Not listed
Enrollment
403 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
37
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Loma Linda, California + 31 more
Source: ClinicalTrials.gov public record
Updated Apr 19, 2026 · Synced Jun 26, 2026, 4:26 AM EDT
Completed Phase 3 Interventional Results available

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

NCT04089566
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
7 Days and older
Enrollment
145 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
7
States / cities
Palo Alto, California • Aurora, Colorado • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2025 · Synced Jun 26, 2026, 4:26 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)

NCT04944940
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy, Kennedys Disease, Motor Neuron Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 120 Years · Male only
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 10, 2026 · Synced Jun 26, 2026, 4:26 AM EDT
Completed No phase listed Observational

Genomic Translation for Amyotrophic Lateral Sclerosis Care

NCT02795897
View directory record Official record
Conditions
ALS
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 80 Years
Enrollment
254 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
13
States / cities
Los Angeles, California • Aurora, Colorado • Ann Arbor, Michigan + 10 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2022 · Synced Jun 26, 2026, 4:26 AM EDT
Completed Phase 3 Interventional Results available

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

NCT03306277
View directory record Official record
Conditions
SMA - Spinal Muscular Atrophy, Gene Therapy
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 180 Days
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
16
States / cities
Los Angeles, California • Stanford, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Jun 26, 2026, 4:26 AM EDT