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ClinicalTrials.gov public records Last synced Oct 21, 2026, 6:52 PM EDT

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Showing 145–164 of 164 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Progressive 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, 6:52 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 21, 2026, 6:52 PM EDT
Active, not recruiting Phase 2 Interventional

Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT06547216
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
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years to 72 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
14
States / cities
Los Angeles, California • Palo Alto, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Oct 21, 2026, 6:52 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 21, 2026, 6:52 PM 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, 6:52 PM 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, 6:52 PM 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, 6:52 PM 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, 6:52 PM 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, 6:52 PM 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, 6:52 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 21, 2026, 6:52 PM 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, 6:52 PM 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, 6:52 PM 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, 6:52 PM 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, 6:52 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 21, 2026, 6:52 PM 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, 6:52 PM 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, 6:52 PM EDT
Completed Phase 3 Interventional Results available

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

NCT03505099
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
onasemnogene abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Up to 42 Days
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
11
States / cities
Los Angeles, California • Aurora, Colorado • Orlando, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2026 · Synced Oct 21, 2026, 6:52 PM EDT
Recruiting Phase 1Phase 2 Interventional

TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC)

NCT07190300
View directory record Official record
Conditions
Metastatic Hormone-Sensitive Prostate Cancer (mHSPC)
Interventions
Tulmimetostat, Darolutamide, Abiraterone, Prednisone/Prednisolone, Androgen Deprivation Therapy (ADT)
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older · Male only
Enrollment
181 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
8
States / cities
Birmingham, Alabama • Iowa City, Iowa • Westwood, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2026 · Synced Oct 21, 2026, 6:52 PM EDT