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Showing 1–24 of 156 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: SMA Clear all
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 Sep 5, 2026, 11:14 PM 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Withdrawn No phase listed Observational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 5, 2026, 11:14 PM 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 5, 2026, 11:14 PM 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 Sep 5, 2026, 11:14 PM EDT
Completed Phase 2 Interventional Results available

Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

NCT02913482
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
1 Month to 7 Months
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
4
States / cities
Palo Alto, California • Chicago, Illinois • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 6, 2024 · Synced Sep 5, 2026, 11:14 PM 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 5, 2026, 11:14 PM 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 5, 2026, 11:14 PM EDT
Completed 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 Aug 10, 2026 · Synced Sep 5, 2026, 11:14 PM EDT
Active, not recruiting Not applicable Interventional

COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis

NCT05035823
View directory record Official record
Conditions
Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis, Muscular Dystrophies, Spinal Cord Injuries, Stroke, Lacunar, Stroke Brainstem, Cervical Spinal Cord Injury, Spinal Muscular Atrophy, Tetraplegic; Paralysis, Quadriplegia/Tetraplegia
Interventions
Motor Neuroprosthesis (MNP)
Device
Lead sponsor
Synchron Medical, Inc.
Industry
Eligibility
21 Years to 75 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
3
States / cities
Buffalo, New York • New York, New York • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 28, 2025 · Synced Sep 5, 2026, 11:14 PM EDT
Completed No phase listed Observational

Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States

NCT06178393
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Up to 5 Years
Enrollment
162 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
1
States / cities
Bannockburn, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 20, 2023 · Synced Sep 5, 2026, 11:14 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

NCT00568698
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Hydroxyurea, Placebo to match hydroxyurea
Drug
Lead sponsor
Stanford University
Other
Eligibility
Up to 2 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2012
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Oct 1, 2019 · Synced Sep 5, 2026, 11:14 PM 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 5, 2026, 11:14 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

NCT02532244
View directory record Official record
Conditions
Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Disease, Peroneal Muscular Atrophy
Interventions
sample collection
Other
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
1 Month and older
Enrollment
230 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2024
U.S. locations
3
States / cities
Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Dec 18, 2024 · Synced Sep 5, 2026, 11:14 PM EDT
Completed No phase listed Observational

Study of ALS Reversals 4: LifeTime Exposures

NCT03706391
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Progressive Muscular Atrophy, Motor Neuron Disease
Interventions
Not listed
Lead sponsor
Duke University
Other
Eligibility
Not listed
Enrollment
25 participants
Timeline
2018 – 2020
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 19, 2023 · Synced Sep 5, 2026, 11:14 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

NCT05089656
View directory record Official record
Conditions
Type 2 Spinal Muscular Atrophy
Interventions
OAV101, Sham control
Genetic · Procedure
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
6
States / cities
Farmington, Connecticut • Chicago, Illinois • Strasburg, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 12, 2026 · Synced Sep 5, 2026, 11:14 PM EDT
Terminated Phase 3 Interventional Results available

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

NCT02193074
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen, Sham procedure
Drug · Procedure
Lead sponsor
Biogen
Industry
Eligibility
Up to 210 Days
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
12
States / cities
Los Angeles, California • Aurora, Colorado • Orlando, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Sep 5, 2026, 11:14 PM EDT
Recruiting Phase 2 Interventional

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 Atrophy

NCT07287982
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
ARGX-119 IV, Placebo IV
Biological · Other
Lead sponsor
argenx
Industry
Eligibility
5 Years to 17 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
16
States / cities
Little Rock, Arkansas • San Diego, California • Stanford, California + 13 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 5, 2026, 11:14 PM EDT
Recruiting No phase listed Observational

Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies

NCT06532474
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
5 Years to 20 Years
Enrollment
24 participants
Timeline
2025 – 2028
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Sep 5, 2026, 11:14 PM EDT
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 5, 2026, 11:14 PM 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 Sep 5, 2026, 11:14 PM EDT
Recruiting Phase 3 Interventional

A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)

NCT07723248
View directory record Official record
Conditions
Metastatic Castration Resistant Prostate Cancer
Interventions
Rinzimetostat, Darolutamide, Enzalutamide, Docetaxel
Drug
Lead sponsor
ORIC Pharmaceuticals
Industry
Eligibility
18 Years and older · Male only
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
7
States / cities
Jeffersonville, Indiana • Charlotte, North Carolina • Canton, Ohio + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Sep 5, 2026, 11:14 PM 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 5, 2026, 11:14 PM 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 5, 2026, 11:14 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Mechanisms of Cell Death in Spinal Muscular Atrophy

NCT01754441
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2020
U.S. locations
2
States / cities
Wilmington, Delaware • Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Feb 20, 2020 · Synced Sep 5, 2026, 11:14 PM EDT