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ClinicalTrials.gov public records Last synced Oct 21, 2026, 9:37 AM EDT

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Showing 169–192 of 194 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Spinal Muscular Atrophy Clear all
Completed Phase 2 Interventional

A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

NCT05808764
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
Up to 19 Days
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
3
States / cities
Chicago, Illinois • Ann Arbor, Michigan • Gordonville, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Oct 21, 2026, 9:37 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 22, 2026 · Synced Oct 21, 2026, 9:37 AM EDT
Recruiting Not applicable Interventional

Nomad P-KAFO Study

NCT05644522
View directory record Official record
Conditions
Cerebrovascular Accident, Post-polio Syndrome, Spinal Cord Injuries, Multiple Sclerosis, Muscular Dystrophy, Paralysis
Interventions
Indego Nomad® Powered Knee-Ankle-Foot Orthosis (P-KAFO) (Parker Hannifin Corp., Cleveland, OH)
Device
Lead sponsor
Shirley Ryan AbilityLab
Other
Eligibility
18 Years to 89 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Oct 21, 2026, 9:37 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 15, 2024 · Synced Oct 21, 2026, 9:37 AM EDT
Active, not recruiting Not applicable Interventional

Comparing Functional Outcomes in Individuals Using Micro-processor Controlled Orthosis Versus Stance Control Orthosis

NCT02089880
View directory record Official record
Conditions
Spinal Cord Injuries, Poliomyelitis, Post-polio Syndrome, Arthritis, Lower Motor Neurone Lesion
Interventions
C-brace, Stance control orthosis
Device
Lead sponsor
Shirley Ryan AbilityLab
Other
Eligibility
18 Years to 80 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated May 1, 2025 · Synced Oct 21, 2026, 9:37 AM 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 21, 2020 · Synced Oct 21, 2026, 9:37 AM EDT
Terminated Phase 2Phase 3 Interventional Results available

Study of the Efficacy and Safety of Immune Globulin Intravenous (Human) Flebogamma® 5% Dual Inactivation and Filtration (DIF) in Participants With Post-polio Syndrome

NCT02176863
View directory record Official record
Conditions
Post-polio Syndrome
Interventions
Flebogamma® 5% DIF, Placebo
Biological · Drug
Lead sponsor
Instituto Grifols, S.A.
Industry
Eligibility
18 Years to 75 Years
Enrollment
191 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
4
States / cities
St Louis, Missouri • Syracuse, New York • Philadelphia, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 5, 2025 · Synced Oct 21, 2026, 9:37 AM EDT
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 15, 2026 · Synced Oct 21, 2026, 9:37 AM EDT
Completed Not applicable Interventional

Power Over Pain (POP) Study

NCT01800604
View directory record Official record
Conditions
Multiple Sclerosis, Spinal Cord Injury, Chronic Pain, Other and Unspecified Amputation Stump Complications, Muscular Dystrophies, Low Back Pain
Interventions
Pain Self-Management Intervention #1, Pain Self-Management Intervention #2, Pain Self-Management Intervention #3, Pain Self-Management Intervention #4
Behavioral
Lead sponsor
University of Washington
Other
Eligibility
18 Years and older
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Oct 17, 2018 · Synced Oct 21, 2026, 9:37 AM EDT
Completed Phase 2 Interventional

Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy

NCT00481013
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Valproic Acid (VPA), Placebo
Drug
Lead sponsor
University of Utah
Other
Eligibility
18 Years to 60 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 6, 2016 · Synced Oct 21, 2026, 9:37 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Clinical, Molecular and Imaging Biomarkers in Spinal and Bulbar Muscular Atrophy (SBMA)

NCT04944940
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy, Kennedys Disease, Motor Neuron Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 120 Years · Male only
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 21, 2026, 9:37 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 19, 2022 · Synced Oct 21, 2026, 9:37 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants

NCT03988907
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam, Midazolam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years to 55 Years
Enrollment
35 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Oct 19, 2020 · Synced Oct 21, 2026, 9:37 AM 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 21, 2026, 9:37 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 15, 2022 · Synced Oct 21, 2026, 9:37 AM EDT
Completed Not applicable Interventional

A Randomized Exercise Trial for Wheelchair Users

NCT00866112
View directory record Official record
Conditions
Spinal Cord Injury, Multiple Sclerosis, Muscular Dystrophy, Cerebral Palsy, Spina Bifida, Amputation, Arthritis, Stroke
Interventions
Educational session to promote physical activity, Minimal contact
Other
Lead sponsor
University of Kansas
Other
Eligibility
18 Years to 65 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
1
States / cities
Lawrence, Kansas
Source: ClinicalTrials.gov public record
Updated Jun 15, 2012 · Synced Oct 21, 2026, 9:37 AM EDT
Recruiting Not applicable Interventional

Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)

NCT06094205
View directory record Official record
Conditions
Anarthria, Dysarthria, Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked-in Syndrome, Muscular Dystrophies
Interventions
BrainGate Neural Interface System
Device
Lead sponsor
Leigh R. Hochberg, MD, PhD.
Other
Eligibility
18 Years to 80 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Dec 1, 2025 · Synced Oct 21, 2026, 9:37 AM 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 17, 2019 · Synced Oct 21, 2026, 9:37 AM EDT
Active, not recruiting Phase 4 Interventional

Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

NCT05232929
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Risdiplam
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
Not listed
Enrollment
403 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
37
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Loma Linda, California + 31 more
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 21, 2026, 9:37 AM EDT
Terminated No phase listed Observational

Infants With Spinal Muscular Atrophy Type I

NCT01547871
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Year to 6 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 12, 2019 · Synced Oct 21, 2026, 9:37 AM 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 21, 2026, 9:37 AM 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 26, 2026 · Synced Oct 21, 2026, 9:37 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 13, 2023 · Synced Oct 21, 2026, 9:37 AM EDT
Completed Not applicable Interventional Results available

ECoG Direct Brain Interface for Individuals With Upper Limb Paralysis

NCT01393444
View directory record Official record
Conditions
Tetraplegia, Spinal Cord Injury, Brachial Plexus Injury, Muscular Dystrophy, ALS, Brainstem Stroke
Interventions
Implantation of ECoG sensors on the brain surface
Device
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 70 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 12, 2016 · Synced Oct 21, 2026, 9:37 AM EDT