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ClinicalTrials.gov public records Last synced Jul 21, 2026, 9:27 AM EDT

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Showing 1–24 of 148 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Spinal Muscular Atrophy (SMA) Clear all
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype and Biomarkers 2

NCT04875416
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Frontotemporal Dementia
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
7 Years and older
Enrollment
217 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2031
U.S. locations
3
States / cities
Miami, Florida • Kansas City, Kansas • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 AM EDT
Completed No phase listed Observational

A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy

NCT07403214
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Not listed
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
4,805 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 10, 2026 · Synced Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study to Evaluate Exicorilant (CORT125281) in Combination With Enzalutamide in Patients With Metastatic Castration-Resistant Prostate Cancer

NCT03437941
View directory record Official record
Conditions
Metastatic Castration-Resistant Prostate Cancer
Interventions
Exicorilant, Enzalutamide, Placebo
Drug
Lead sponsor
Corcept Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
6
States / cities
Scottsdale, Arizona • Detroit, Michigan • Basking Ridge, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

COVID-19 In-vitro Diagnostic Test and Androgen Receptor Gene Expression

NCT04530500
View directory record Official record
Conditions
Covid19, Androgen Receptor Gene Overexpression
Interventions
COVID-19 Androgen Sensitivity Test (CoVAST)
Genetic
Lead sponsor
University of California, Irvine
Other
Eligibility
18 Years to 90 Years · Male only
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
2
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated May 7, 2024 · Synced Jul 21, 2026, 9:27 AM EDT
Active, not recruiting Phase 3 Interventional

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

NCT05626855
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
Drug
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years and older
Enrollment
238 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
25
States / cities
Phoenix, Arizona • La Jolla, California • Los Angeles, California + 22 more
Source: ClinicalTrials.gov public record
Updated May 24, 2025 · Synced Jul 21, 2026, 9:27 AM 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 Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 AM EDT
Completed Phase 3 Interventional Results available

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)

NCT02292537
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Nusinersen, Sham procedure
Drug · Procedure
Lead sponsor
Biogen
Industry
Eligibility
2 Years to 12 Years
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
11
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 AM EDT
Terminated Phase 1 Interventional

A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)

NCT02240355
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
RO6885247, placebo
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
Up to 55 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
6
States / cities
Stanford, California • Orlando, Florida • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 21, 2016 · Synced Jul 21, 2026, 9:27 AM 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 Jul 21, 2026, 9:27 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

NCT04798378
View directory record Official record
Conditions
Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies, Arthrogryposis, Spinal Cord Injuries, Charcot-Marie-Tooth, Stroke, Weakness of Extremities as Sequela of Stroke, Weakness Due to Upper Motor Neuron Dysfunction, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, Arm Paralysis, Cerebral Palsy
Interventions
Neurosleeve
Device
Lead sponsor
Thomas Jefferson University
Other
Eligibility
4 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
2
States / cities
Wilmington, Delaware • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 8, 2025 · Synced Jul 21, 2026, 9:27 AM EDT
Completed Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

NCT05517603
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
Interventions
AJ201, Placebo
Drug
Lead sponsor
AnnJi Pharmaceutical Co., Ltd.
Industry
Eligibility
18 Years and older · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
6
States / cities
Orange, California • Palo Alto, California • Jacksonville, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2024 · Synced Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 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 Jul 21, 2026, 9:27 AM EDT
Completed No phase listed Observational

Measuring Levels of SMN in Blood Samples of SMA Patients

NCT00061607
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
2 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2017 · Synced Jul 21, 2026, 9:27 AM EDT
Completed Phase 2 Interventional Results available

Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy

NCT00227266
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Valproic Acid and Levocarnitine, Placebo
Drug
Lead sponsor
University of Utah
Other
Eligibility
2 Years to 17 Years
Enrollment
94 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
5
States / cities
Baltimore, Maryland • Detroit, Michigan • Columbus, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Jul 21, 2026, 9:27 AM EDT