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ClinicalTrials.gov public records Last synced Oct 21, 2026, 3:41 AM EDT

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Showing 145–168 of 194 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Spinal Muscular Atrophy Clear all
Recruiting Phase 2 Interventional

Vasodilator and Exercise Study for DMD (VASO-REx)

NCT06290713
View directory record Official record
Conditions
Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy, Muscular Dystrophy in Children, Vasodilation, Exercise, DMD
Interventions
Tadalafil, Placebo, Exercise Training
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
6 Years and older · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 21, 2026, 3:41 AM EDT
Recruiting Not applicable Interventional

Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia

NCT05724173
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
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
2
States / cities
Stanford, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Oct 21, 2026, 3:41 AM EDT
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 9, 2010 · Synced Oct 21, 2026, 3:41 AM 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, 3:41 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, 3:41 AM 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 17, 2021 · Synced Oct 21, 2026, 3:41 AM 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, 3:41 AM 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 Oct 8, 2026 · Synced Oct 21, 2026, 3:41 AM EDT
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, 3:41 AM 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, 3:41 AM 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 21, 2026, 3:41 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, 3:41 AM 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 21, 2026, 3:41 AM 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 21, 2026, 3:41 AM EDT
Terminated Phase 3 Interventional Results available

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

NCT02193074
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
nusinersen, Sham procedure
Drug · Procedure
Lead sponsor
Biogen
Industry
Eligibility
Up to 210 Days
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
12
States / cities
Los Angeles, California • Aurora, Colorado • Orlando, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2021 · Synced Oct 21, 2026, 3:41 AM EDT
Completed Phase 4 Interventional

Myobloc Atrophy Study

NCT02052024
View directory record Official record
Conditions
Spasticity Secondary to Either a Disorder or Trauma, Spinal Cord Injury (SCI), Brain Injury, Tumor, Stroke
Interventions
Botox, MYOBLOC
Drug
Lead sponsor
Georgetown University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Dec 16, 2015 · Synced Oct 21, 2026, 3:41 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
View directory record Official record
Conditions
All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
17,667 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Oct 21, 2026, 3:41 AM EDT
Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

NCT05337553
View directory record Official record
Conditions
Spinal Muscular Atrophy, Neuromuscular Diseases, SMA
Interventions
taldefgrobep alfa, Placebo
Drug
Lead sponsor
Biohaven Pharmaceuticals, Inc.
Industry
Eligibility
4 Years to 21 Years
Enrollment
269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
30
States / cities
Phoenix, Arizona • La Jolla, California • Los Angeles, California + 27 more
Source: ClinicalTrials.gov public record
Updated Jun 1, 2026 · Synced Oct 21, 2026, 3:41 AM 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, 3:41 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Brain Physiology in Polio Survivors

NCT00080600
View directory record Official record
Conditions
Postpoliomyelitis
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
21 Years to 80 Years
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2007
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 21, 2026, 3:41 AM EDT
Approved For Marketing No phase listed Expanded access

An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

NCT04256265
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Risdiplam
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
2 Months and older
U.S. locations
29
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 25 more
Source: ClinicalTrials.gov public record
Updated Oct 5, 2020 · Synced Oct 21, 2026, 3:41 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

PatientSpot Formerly Known as ArthritisPower

NCT03840928
View directory record Official record
Conditions
Rheumatoid Arthritis, Ankylosing Spondylitis, Fibromyalgia, Gout, Crohn Disease, Juvenile Idiopathic Arthritis, Lupus Erythematosus, Myositis, Osteoarthritis, Osteoporosis, Psoriasis, Psoriatic Arthritis, Scleroderma, Dermatomyositis, Inflammatory Bowel Diseases, Polymyositis, Axial Spondyloarthritis, Diffuse Idiopathic Skeletal Hyperostosis, Polymyalgia Rheumatica, Giant Cell Arteritis, Temporal Arteritis, Wegener, Relapsing Polychondritis, Undifferentiated Connective Tissue Disease, Spinal Cord Injuries, Alzheimer Disease, Amyotrophic Lateral Sclerosis, Ataxia, Bell Palsy, Brain Tumor, Cerebral Aneurysm, Epilepsy, Guillain-Barre Syndrome, Headache, Head Injury, Hydrocephalus, Lumbar Disc Disease, Meningitis, Multiple Sclerosis, Muscular Dystrophy, Neurocutaneous Syndromes, Parkinson Disease, Stroke, Cluster Headache, Tension-Type Headache, Chronic Obstructive Pulmonary Disease, Asthma, Lung Cancer, Cystic Fibrosis, Sleep Apnea, Eczema, Alopecia, Chronic Inflammation, Unstable Angina, Heart Attack, Heart Failure, Arrythmia, Valve Heart Disease, High Blood Pressure, Congenital Heart Disease, Peripheral Arterial Disease, Diabetes, Chronic Liver Disease, Obesity
Interventions
Not listed
Lead sponsor
Global Healthy Living Foundation
Other
Eligibility
19 Years and older
Enrollment
40,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
1
States / cities
Upper Nyack, New York
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Oct 21, 2026, 3:41 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Biofluid Research on Age-Related or Inherited Neurodegeneration

NCT07798700
View directory record Official record
Conditions
Frontotemporal Degeneration (FTD), ALS (Amyotrophic Lateral Sclerosis), Primary Progressive Aphasia (PPA), Familial Frontotemporal Lobar Degeneration (fFTLD), Fronto-temporal Lobar Dementia, Dementia With Lewy Bodies (DLB), Cortical Basal Syndrome (CBS), Alzheimer's Disease (AD), Progressive Supranuclear Palsy(PSP), Parkinson's Disease (PD), Parkinson's Disease Dementia (PDD), Limbic-predominant Age-related TDP-43 Encephalopathy (LATE), Posterior Cortical Atrophy (PCA), Primary Lateral Sclerosis (PLS), Primary Muscular Atrophy (PMA)
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2070
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 1, 2026 · Synced Oct 21, 2026, 3:41 AM EDT
Terminated Phase 1 Interventional Results available

Study to Compare Capsule and Liquid Formulations of Enzalutamide After Single Dose Administration Under Fasting Conditions in Prostate Cancer

NCT03478904
View directory record Official record
Conditions
Prostate Cancer
Interventions
Enzalutamide Liquid, Enzalutamide Capsule
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 10, 2021 · Synced Oct 21, 2026, 3:41 AM EDT