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Showing 1–24 of 151 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Spinal Muscular Atrophy (SMA) Clear all
Terminated Phase 2 Interventional

A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.

NCT03196375
View directory record Official record
Conditions
Motor Neuron Disease
Interventions
FLX-787-ODT (orally disintegrating tablet), Placebo ODT
Drug
Lead sponsor
Flex Pharma, Inc.
Industry
Eligibility
18 Years and older
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
35
States / cities
Scottsdale, Arizona • Sacramento, California • San Francisco, California + 29 more
Source: ClinicalTrials.gov public record
Updated Aug 28, 2018 · Synced Sep 15, 2026, 9:49 AM EDT
Completed Phase 1 Interventional

An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)

NCT01494701
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 14 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Sep 15, 2026, 9:49 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 Sep 15, 2026, 9:49 AM EDT
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 15, 2026, 9:49 AM EDT
Completed No phase listed Observational

Study of Hepatic Function in Patients With Spinal and Bulbar Muscular Atrophy

NCT02124057
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy (SBMA), Motor Neuron Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 17, 2022 · Synced Sep 15, 2026, 9:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Study of ALS Reversals 2: Genetic Analyses

NCT03464903
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
26 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 10, 2024 · Synced Sep 15, 2026, 9:49 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 Sep 15, 2026, 9:49 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 15, 2026, 9:49 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 15, 2026, 9:49 AM EDT
Recruiting Phase 3 Interventional

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 Risdiplam

NCT07444476
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Salanersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
15 Years to 60 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Sep 15, 2026, 9:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)

NCT04139343
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Ohio State University
Other
Eligibility
17 Years to 70 Years
Enrollment
67 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2025
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 22, 2026 · Synced Sep 15, 2026, 9:49 AM EDT
Completed Phase 3 Interventional

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

NCT05156320
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
Interventions
Apitegromab, Placebo
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
26
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Sep 15, 2026, 9:49 AM EDT
Completed No phase listed Observational

Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain

NCT06632730
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Interventions
Not listed
Lead sponsor
Novartis
Industry
Eligibility
Up to 14 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 8, 2024 · Synced Sep 15, 2026, 9:49 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy Studies of Panobinostat and Bicalutamide in Patients With Recurrent Prostate Cancer After Castration

NCT00878436
View directory record Official record
Conditions
Prostate Cancer, Prostatic Neoplasms
Interventions
Panobinostat, Bicalutamide
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older · Male only
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
4
States / cities
New Brunswick, New Jersey • Lake Success, New York • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2018 · Synced Sep 15, 2026, 9:49 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

NCT06839469
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Sep 15, 2026, 9:49 AM EDT
Withdrawn Early Phase 1 Interventional

Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments

NCT04050852
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen Treatments
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
5 Years to 21 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 28, 2021 · Synced Sep 15, 2026, 9:49 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy

NCT02024932
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy
Interventions
BVS857, Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older · Male only
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
3
States / cities
Orange, California • Bethesda, Maryland • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 4, 2021 · Synced Sep 15, 2026, 9:49 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 15, 2026, 9:49 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 15, 2026, 9:49 AM EDT
Completed Phase 3 Interventional Results available

A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies

NCT02594124
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
Not listed
Enrollment
292 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
18
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 15 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2024 · Synced Sep 15, 2026, 9:49 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 15, 2026, 9:49 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

NCT04718181
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
risdiplam, omeprazole
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 55 Years
Enrollment
131 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2023
U.S. locations
4
States / cities
Daytona Beach, Florida • Springfield, Missouri • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2023 · Synced Sep 15, 2026, 9:49 AM EDT
Withdrawn No phase listed Observational

Multi-disease Carrier Screening Test Validation

NCT01663584
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA), Carrier Screening, Genetic Testing
Interventions
Blood draw
Procedure
Lead sponsor
Natera, Inc.
Industry
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
San Carlos, California
Source: ClinicalTrials.gov public record
Updated Jul 15, 2013 · Synced Sep 15, 2026, 9:49 AM EDT
Completed Phase 1 Interventional Results available

Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1

NCT02122952
View directory record Official record
Conditions
Spinal Muscular Atrophy 1
Interventions
AVXS-101
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 6 Months
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 14, 2022 · Synced Sep 15, 2026, 9:49 AM EDT