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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Progressive Muscular Atrophy Clear all
Completed No phase listed Observational

Measuring Levels of SMN in Blood Samples of SMA Patients

NCT00061607
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
2 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2017 · Synced Jul 22, 2026, 9:46 PM EDT
Recruiting Not applicable Interventional

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

NCT07521930
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 22, 2026, 9:46 PM 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 17, 2024 · Synced Jul 22, 2026, 9:46 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 Jul 22, 2026, 9:46 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients

NCT00568802
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Hydroxyurea, Placebo to match hydroxyurea
Drug
Lead sponsor
Stanford University
Other
Eligibility
1 Year to 10 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Stanford, California
Source: ClinicalTrials.gov public record
Updated Oct 1, 2019 · Synced Jul 22, 2026, 9:46 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

NCT02327845
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Progressive Muscular Atrophy, Multisystem Proteinopathy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
Not listed
Enrollment
708 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2031
U.S. locations
13
States / cities
Palo Alto, California • San Diego, California • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Jul 22, 2026, 9:46 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 14, 2025 · Synced Jul 22, 2026, 9:46 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 19, 2023 · Synced Jul 22, 2026, 9:46 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
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 Jun 22, 2026 · Synced Jul 22, 2026, 9:46 PM EDT
Completed Phase 2 Interventional Results available

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

NCT02908685
View directory record Official record
Conditions
Muscular Atrophy, Spinal
Interventions
Placebo, Risdiplam
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years to 25 Years
Enrollment
231 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
2
States / cities
Palo Alto, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 23, 2024 · Synced Jul 22, 2026, 9:46 PM EDT
Withdrawn No phase listed Observational

Breast Cancer BRCA1 Carriers: a Pilot Study

NCT05062174
View directory record Official record
Conditions
BRCA1 Mutation, High-grade Serous Ovarian Cancer, TNBC - Triple-Negative Breast Cancer
Interventions
Mifepristone 200 MG, Prophylactic mastectomy
Drug · Procedure
Lead sponsor
Indiana University
Other
Eligibility
18 Years and older · Female only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
2
States / cities
Carmel, Indiana • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Dec 14, 2022 · Synced Jul 22, 2026, 9:46 PM EDT
Recruiting No phase listed Observational

Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA

NCT06955897
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
12 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced Jul 22, 2026, 9:46 PM EDT
Completed Not applicable Interventional

Effects of Standing on Non-Ambulatory Children With Spinal Muscular Atrophy

NCT02341053
View directory record Official record
Conditions
Spinal Muscular Dystrophy, Neuromuscular Disability
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 16, 2021 · Synced Jul 22, 2026, 9:46 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 Jul 22, 2026, 9:46 PM 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 16, 2021 · Synced Jul 22, 2026, 9:46 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 Jul 22, 2026, 9:46 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 Jul 22, 2026, 9:46 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 13, 2017 · Synced Jul 22, 2026, 9:46 PM EDT
Active, not recruiting Phase 3 Interventional

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

NCT05335876
View directory record Official record
Conditions
Spinal Muscular Atrophy (SMA)
Interventions
onasemnogene abeparvovec
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
0 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2031
U.S. locations
1
States / cities
Norfolk, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Jul 22, 2026, 9:46 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

NCT06839469
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD)
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2028
U.S. locations
3
States / cities
Palo Alto, California • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Jul 22, 2026, 9:46 PM 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 4, 2020 · Synced Jul 22, 2026, 9:46 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 Jul 5, 2026 · Synced Jul 22, 2026, 9:46 PM 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 21, 2026 · Synced Jul 22, 2026, 9:46 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 Jul 22, 2026, 9:46 PM EDT