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ClinicalTrials.gov public records Last synced Oct 21, 2026, 12:05 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 158 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: SMA Clear all
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 21, 2026, 12:05 PM EDT
Completed No phase listed Observational

International SMA Patient Registry

NCT00466349
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Not listed
Lead sponsor
Indiana University
Other
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1986 – 2018
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Aug 7, 2018 · Synced Oct 21, 2026, 12:05 PM EDT
Recruiting Phase 4 Interventional

ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)

NCT07448610
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Onasemnogene Abeparvovec-brve
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 100 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 21, 2026, 12:05 PM EDT
Completed Phase 3 Interventional Results available

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

NCT05386680
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Norfolk, Virginia • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 21, 2026, 12:05 PM EDT
Active, not recruiting Not applicable Interventional

Pediatric Powered Wheelchair Standing Devices: An Exploratory Study

NCT05117827
View directory record Official record
Conditions
Cerebral Palsy, Spinal Muscular Atrophy, Spinal Cord Injuries, Spina Bifida, Development Delay, Genetic Disease
Interventions
Power Wheelchair Standing Device
Device
Lead sponsor
Grand Valley State University
Other
Eligibility
5 Years to 17 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Grand Rapids, Michigan
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 21, 2026, 12:05 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 9, 2026 · Synced Oct 21, 2026, 12:05 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 20, 2023 · Synced Oct 21, 2026, 12:05 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 16, 2013 · Synced Oct 21, 2026, 12:05 PM EDT
Recruiting No phase listed Observational

A Registry of Cases of Spinal Muscular Atrophy

NCT07805278
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
30 participants
Timeline
2026 – 2033
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 30, 2026 · Synced Oct 21, 2026, 12:05 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 21, 2023 · Synced Oct 21, 2026, 12:05 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 14, 2017 · Synced Oct 21, 2026, 12:05 PM 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 10, 2023 · Synced Oct 21, 2026, 12:05 PM 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 8, 2024 · Synced Oct 21, 2026, 12:05 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 21, 2026, 12:05 PM EDT
Completed No phase listed Observational

Adults With SMA Treated With Nusinersen

NCT04591678
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Ohio State University
Other
Eligibility
18 Years to 60 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 22, 2021 · Synced Oct 21, 2026, 12:05 PM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Gene Therapy for IGHMBP2-Related Diseases

NCT05152823
View directory record Official record
Conditions
SMARD1, CMT2S
Interventions
Gene Therapy
Biological
Lead sponsor
Megan Waldrop
Other
Eligibility
2 Months to 14 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 30, 2025 · Synced Oct 21, 2026, 12:05 PM 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 18, 2022 · Synced Oct 21, 2026, 12:05 PM EDT
Completed No phase listed Observational

A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis

NCT01910168
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Not listed
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
Not listed
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
2
States / cities
Kalamazoo, Michigan • Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Nov 2, 2016 · Synced Oct 21, 2026, 12:05 PM EDT
Completed Not applicable Interventional Results available

Spinal Cord Stimulation in Spinal Muscular Atrophy

NCT05430113
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 4
Interventions
Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)
Device
Lead sponsor
Marco Capogrosso
Other
Eligibility
16 Years to 64 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Oct 21, 2026, 12:05 PM EDT
Not listed Phase 4 Interventional

Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

NCT05522361
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam
Drug
Lead sponsor
Clinic for Special Children
Other
Eligibility
2 Years to 35 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Strasburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 21, 2026, 12:05 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Oxidative Capacity and Exercise Tolerance in Ambulatory SMA

NCT02895789
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
8 Years to 55 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 8, 2022 · Synced Oct 21, 2026, 12:05 PM 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
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 21, 2026, 12:05 PM 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 Sep 1, 2026 · Synced Oct 21, 2026, 12:05 PM 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 22, 2016 · Synced Oct 21, 2026, 12:05 PM EDT