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Showing 1–24 of 161 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Muscular Atrophy, Progressive Clear all
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

NCT00439218
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Months to 48 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
5
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 8, 2010 · Synced Sep 1, 2026, 5:56 PM 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 Sep 1, 2026, 5:56 PM EDT
Completed Not applicable Interventional

Study of Resistance Strengthening Versus Ambulation Post-Liver Transplant

NCT01216722
View directory record Official record
Conditions
Liver Transplantation, Resistance Training, Activities of Daily Living
Interventions
Resistance Strengthening Exercise Program
Other
Lead sponsor
University of Miami
Other
Eligibility
18 Years to 80 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Coral Gables, Florida
Source: ClinicalTrials.gov public record
Updated Oct 6, 2010 · Synced Sep 1, 2026, 5:56 PM 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 Sep 1, 2026, 5:56 PM EDT
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 30, 2020 · Synced Sep 1, 2026, 5:56 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 1, 2026, 5:56 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 1, 2016 · Synced Sep 1, 2026, 5:56 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 1, 2026, 5:56 PM EDT
Recruiting No phase listed Observational

ALS/MND Natural History Study Data Repository

NCT05966038
View directory record Official record
Conditions
ALS, PLS, MND (Motor Neurone DIsease), Kennedy Disease, PMA - Progressive Muscular Atrophy, PBP - Progressive Bulbar Palsy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2030
U.S. locations
14
States / cities
Loma Linda, California • Los Angeles, California • Gainesville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced Sep 1, 2026, 5:56 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 1, 2026, 5:56 PM EDT
Not listed Not applicable Interventional

Schulze Muscular Dystrophy Ability Clinical Study

NCT05409079
View directory record Official record
Conditions
Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy, FSHD, Cerebral Palsy, Becker Muscular Dystrophy
Interventions
Abilitech Assist
Device
Lead sponsor
AbiliTech Medical Inc.
Industry
Eligibility
10 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 4, 2022 · Synced Sep 1, 2026, 5:56 PM 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 Sep 1, 2026, 5:56 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy Studies of Panobinostat and Bicalutamide in Patients With Recurrent Prostate Cancer After Castration

NCT00878436
View directory record Official record
Conditions
Prostate Cancer, Prostatic Neoplasms
Interventions
Panobinostat, Bicalutamide
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older · Male only
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
4
States / cities
New Brunswick, New Jersey • Lake Success, New York • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 6, 2018 · Synced Sep 1, 2026, 5:56 PM EDT
Completed Phase 1 Interventional

An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)

NCT02052791
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
Not listed
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
4
States / cities
Boston, Massachusetts • New York, New York • Dallas, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 15, 2021 · Synced Sep 1, 2026, 5:56 PM EDT
Recruiting Phase 3 Interventional

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam

NCT07444476
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Salanersen
Drug
Lead sponsor
Biogen
Industry
Eligibility
15 Years to 60 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Sep 1, 2026, 5:56 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 Aug 12, 2026 · Synced Sep 1, 2026, 5:56 PM EDT
Completed Phase 3 Interventional Results available

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

NCT03306277
View directory record Official record
Conditions
SMA - Spinal Muscular Atrophy, Gene Therapy
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 180 Days
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
16
States / cities
Los Angeles, California • Stanford, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2026 · Synced Sep 1, 2026, 5:56 PM 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 Sep 1, 2026, 5:56 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs

NCT07336446
View directory record Official record
Conditions
Prostate Cancer
Interventions
AZD9750, AZD5305
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years and older · Male only
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
8
States / cities
Duarte, California • San Francisco, California • Tampa, Florida + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2026 · Synced Sep 1, 2026, 5:56 PM EDT
Completed Phase 1Phase 2 Interventional

Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT05747924
View directory record Official record
Conditions
FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH
Interventions
AOC 1020, Placebo
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years to 70 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
14
States / cities
Los Angeles, California • Palo Alto, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 15, 2026 · Synced Sep 1, 2026, 5:56 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Wearable Technology to Assess Gait Function in SMA and DMD

NCT04193085
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 22, 2024 · Synced Sep 1, 2026, 5:56 PM EDT
Recruiting Phase 4 Interventional

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

NCT05861986
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
3 Months to 24 Months
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
11
States / cities
Little Rock, Arkansas • Aurora, Colorado • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Sep 1, 2026, 5:56 PM EDT
Completed Phase 2 Interventional Results available

An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

NCT03921528
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
Interventions
SRK-015
Biological
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2024 · Synced Sep 1, 2026, 5:56 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 9, 2023 · Synced Sep 1, 2026, 5:56 PM EDT