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ClinicalTrials.gov public records Last synced Oct 22, 2026, 12:40 PM EDT

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Showing 1–24 of 153 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: SMA - Spinal Muscular Atrophy Clear all
Completed Phase 2 Interventional Results available

A Study of CK-2127107 in Patients With Spinal Muscular Atrophy

NCT02644668
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Placebo, Reldesemtiv 150 mg, Reldesemtiv 450 mg
Drug
Lead sponsor
Cytokinetics
Industry
Eligibility
12 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
15
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2020 · Synced Oct 22, 2026, 12:40 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Androgen Receptor, Implications for Health and Wellbeing: Natural History Study of Individuals With Androgen Insensitivity

NCT04708431
View directory record Official record
Conditions
Androgen Insensitivity Syndrome, Metabolic Parameters in AIS, CAIS, PAIS and MAIS, Tumor Formation in AIS, CAIS, PAIS and MAIS, Sexual Function AIS, CAIS, PAIS and MAIS
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
8 Years to 99 Years
Enrollment
650 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2040
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 20, 2026 · Synced Oct 22, 2026, 12:40 PM EDT
Completed No phase listed Observational

Effect of Nusinersen on Adults With Spinal Muscular Atrophy

NCT03878030
View directory record Official record
Conditions
Adult Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Northwell Health
Other
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Great Neck, New York
Source: ClinicalTrials.gov public record
Updated Mar 20, 2023 · Synced Oct 22, 2026, 12:40 PM EDT
Recruiting Phase 1 Interventional

A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)

NCT06555419
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Nusinersen, ThecaFlex DRx System
Drug · Device
Lead sponsor
Biogen
Industry
Eligibility
3 Years and older
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
12
States / cities
Phoenix, Arizona • Orange, California • Palo Alto, California + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 22, 2026, 12:40 PM 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 15, 2024 · Synced Oct 22, 2026, 12:40 PM 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 Oct 21, 2026 · Synced Oct 22, 2026, 12:40 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 19, 2024 · Synced Oct 22, 2026, 12:40 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer

NCT06764485
View directory record Official record
Conditions
Metastatic Castration-resistant Prostate Cancer
Interventions
BMS-986365, Enzalutamide, Abiraterone, Docetaxel, Predinsone/Prednisolone
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older · Male only
Enrollment
960 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
48
States / cities
Birmingham, Alabama • Gilbert, Arizona • Anaheim, California + 34 more
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 22, 2026, 12:40 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development

NCT01736553
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
Not listed
Lead sponsor
Ohio State University
Other
Eligibility
Up to 6 Months
Enrollment
53 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
15
States / cities
Davis, California • Los Angeles, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated May 4, 2018 · Synced Oct 22, 2026, 12:40 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

NCT05794139
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
NMD670, Placebo
Drug
Lead sponsor
NMD Pharma A/S
Industry
Eligibility
18 Years to 75 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
9
States / cities
Los Angeles, California • Palo Alto, California • Kansas City, Kansas + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 22, 2026, 12:40 PM 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 31, 2025 · Synced Oct 22, 2026, 12:40 PM EDT
Completed Phase 1 Interventional

Patients With ALS and Other Motor Disorders Will be Treated With Umbilical or Placental Mesenchymal Cell Exosome Solution

NCT07105371
View directory record Official record
Conditions
Motor Disorders
Interventions
Exosome Secretome
Biological
Lead sponsor
The Foundation for Orthopaedics and Regenerative Medicine
Other
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Naples, Florida
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 22, 2026, 12:40 PM EDT
Completed Phase 1Phase 2 Interventional Results available

CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

NCT00661453
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I
Interventions
Valproic Acid and Levocarnitine
Drug
Lead sponsor
University of Utah
Other
Eligibility
2 Weeks to 12 Months
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
6
States / cities
Baltimore, Maryland • Detroit, Michigan • Durham, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2015 · Synced Oct 22, 2026, 12:40 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients

NCT00568802
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Hydroxyurea, Placebo to match hydroxyurea
Drug
Lead sponsor
Stanford University
Other
Eligibility
1 Year to 10 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Oct 2, 2019 · Synced Oct 22, 2026, 12:40 PM 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 15, 2026 · Synced Oct 22, 2026, 12:40 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 13, 2026 · Synced Oct 22, 2026, 12:40 PM 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 20, 2025 · Synced Oct 22, 2026, 12:40 PM 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 28, 2025 · Synced Oct 22, 2026, 12:40 PM 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 9, 2025 · Synced Oct 22, 2026, 12:40 PM 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 22, 2026 · Synced Oct 22, 2026, 12:40 PM EDT
Recruiting No phase listed Observational

Diagnostic Journey, Patient Experience, and Disparities in the Treatment of Spinal Muscular Atrophy (SMA) in the MedStar Health System

NCT07223489
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
Telephone Interview
Behavioral
Lead sponsor
Medstar Health Research Institute
Other
Eligibility
18 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Washinton, District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 22, 2026, 12:40 PM 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 8, 2010 · Synced Oct 22, 2026, 12:40 PM EDT
Completed Phase 2 Interventional

Effects of Power Mobility on Young Children With Severe Motor Impairments

NCT01028833
View directory record Official record
Conditions
Cerebral Palsy, Arthrogryposis, Spinal Muscular Atrophy, Other Central Nervous System or Musculoskeletal Disorders
Interventions
Power mobility
Other
Lead sponsor
University of Oklahoma
Other
Eligibility
14 Months to 30 Months
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Oklahoma City, Oklahoma
Source: ClinicalTrials.gov public record
Updated Jan 14, 2016 · Synced Oct 22, 2026, 12:40 PM 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 29, 2026 · Synced Oct 22, 2026, 12:40 PM EDT