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ClinicalTrials.gov public records Last synced Oct 4, 2026, 11:10 AM EDT

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Showing 25–48 of 910 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neuromuscular; Disorder, Hereditary Clear all
Recruiting Phase 2 Interventional

A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY)

NCT07175038
View directory record Official record
Conditions
Pulmonary Arterial Hypertension (PAH), Pulmonary Hypertension, Interstitial Lung Disease
Interventions
ROC-101
Drug
Lead sponsor
AllRock Bio, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
11
States / cities
Phoenix, Arizona • Rancho Cordova, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 10, 2026 · Synced Oct 4, 2026, 11:10 AM EDT
Completed Not applicable Interventional

Clinical Trial of a Serious Game for Individuals With SCI/D

NCT02341950
View directory record Official record
Conditions
Spinal Cord Injury, Spinal Cord Involvement, Spina Bifida, Transverse Myelitis, Polio and Post-polio Syndrome, Syringomyelia, Spinal Stenosis, Spinal Neoplasms, Spinal Cord Diseases
Interventions
SCI Hard
Behavioral
Lead sponsor
University of Michigan
Other
Eligibility
13 Years to 29 Years
Enrollment
184 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Feb 22, 2019 · Synced Oct 4, 2026, 11:10 AM EDT
Recruiting No phase listed Observational

Molecular Analysis of Patients With Neuromuscular Disease

NCT00390104
View directory record Official record
Conditions
Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy, Limb-girdle Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Week to 100 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 24, 2023 · Synced Oct 4, 2026, 11:10 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 4, 2026, 11:10 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 4, 2026, 11:10 AM EDT
Completed No phase listed Observational

Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain

NCT06632730
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Interventions
Not listed
Lead sponsor
Novartis
Industry
Eligibility
Up to 14 Years
Enrollment
69 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
East Hanover, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 9, 2024 · Synced Oct 4, 2026, 11:10 AM EDT
Active, not recruiting Phase 1 Interventional

EGFR806 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults

NCT03618381
View directory record Official record
Conditions
Pediatric Solid Tumor, Germ Cell Tumor, Retinoblastoma, Hepatoblastoma, Wilms Tumor, Rhabdoid Tumor, Carcinoma, Osteosarcoma, Ewing Sarcoma, Rhabdomyosarcoma, Synovial Sarcoma, Clear Cell Sarcoma, Malignant Peripheral Nerve Sheath Tumors, Desmoplastic Small Round Cell Tumor, Soft Tissue Sarcoma, Neuroblastoma
Interventions
second generation 4-1BBζ EGFR806-EGFRt, second generation 4-1BBζ EGFR806-EGFRt and a second generation 4 1BBζ CD19-Her2tG
Biological
Lead sponsor
Seattle Children's Hospital
Other
Eligibility
1 Year to 30 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2040
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Oct 4, 2026, 11:10 AM EDT
Completed Phase 2 Interventional Results available

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

NCT03796637
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ataluren
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 5, 2022 · Synced Oct 4, 2026, 11:10 AM EDT
Completed No phase listed Observational

Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy

NCT01451281
View directory record Official record
Conditions
Muscular Dystrophy, Muscular Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
5 Years to 17 Years · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 22, 2019 · Synced Oct 4, 2026, 11:10 AM EDT
Completed Not applicable Interventional Results available

Innovation in the Treatment of Persistent Pain in Adults With NF1: Implementation of the iCanCope Mobile Application- Clinical Trial

NCT04561765
View directory record Official record
Conditions
Neurofibromatosis 1, Chronic Pain
Interventions
iCanCope, iCanCope+Contingency Management
Device
Lead sponsor
Yale University
Other
Eligibility
18 Years to 72 Years
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 30, 2025 · Synced Oct 4, 2026, 11:10 AM EDT
Completed Not applicable Interventional Results available

Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy

NCT01645098
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Ketamine, Dexmedetomidine
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Male only
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Feb 26, 2015 · Synced Oct 4, 2026, 11:10 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 4, 2026, 11:10 AM EDT
Completed No phase listed Observational

Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

NCT00312247
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
4 Years to 21 Years · Male only
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
3
States / cities
Los Angeles, California • Sacramento, California • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 19, 2015 · Synced Oct 4, 2026, 11:10 AM EDT
Completed Phase 1 Interventional

Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy

NCT02362438
View directory record Official record
Conditions
Giant Axonal Neuropathy, Gene Transfer
Interventions
scAAv9/JeT-GAN
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
3 Years to 99 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 4, 2026, 11:10 AM EDT
Active, not recruiting No phase listed Observational

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

NCT03458832
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
FSHD-specific functional rating scale, Electrical Impedance Myography
Diagnostic Test · Device
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
324 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
8
States / cities
Los Angeles, California • Kansas City, Kansas • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 4, 2026, 11:10 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Neurofibromatosis Type 1 Patient Registry

NCT01410006
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
Not listed
Enrollment
2,391 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2017
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 25, 2017 · Synced Oct 4, 2026, 11:10 AM EDT
Completed Phase 2 Interventional Results available

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

NCT01540409
View directory record Official record
Conditions
Duchenne Muscular Dystrophy (DMD)
Interventions
AVI-4658 (Eteplirsen)
Drug
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
7 Years to 13 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
11
States / cities
Long Beach, California • Gainesville, Florida • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2020 · Synced Oct 4, 2026, 11:10 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 4, 2026, 11:10 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study of the Experimental Drug BKM120 With Paclitaxel in Patients With HER2 Negative, Locally Advanced or Metastatic Breast Cancer, With or Without PI3K Activation

NCT01572727
View directory record Official record
Conditions
Breast Cancer
Interventions
Paclitaxel, BKM120 matching placebo, BKM120
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older · Female only
Enrollment
416 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
21
States / cities
Chandler, Arizona • Phoenix, Arizona • Fayetteville, Arkansas + 17 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2017 · Synced Oct 4, 2026, 11:10 AM EDT
Terminated Phase 1 Interventional

A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74

NCT06597656
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, Plasmapheresis
Genetic · Procedure
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 8 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
3
States / cities
Gainesville, Florida • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 4, 2025 · Synced Oct 4, 2026, 11:10 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Early Detection of Neuropathy in ATTRv

NCT05311488
View directory record Official record
Conditions
Hereditary Amyloidosis, Transthyretin-Related
Interventions
neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
Diagnostic Test
Lead sponsor
University of Pennsylvania
Other
Eligibility
30 Years to 90 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 4, 2026, 11:10 AM EDT
Terminated Phase 2 Interventional Results available

Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)

NCT02927080
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy
Interventions
ACE-083, ACE-083 or placebo
Drug
Lead sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Industry
Eligibility
18 Years and older
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
18
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 15 more
Source: ClinicalTrials.gov public record
Updated Sep 26, 2022 · Synced Oct 4, 2026, 11:10 AM EDT
Recruiting Phase 2 Interventional

A Study of Selinexor in People With Wilms Tumors and Other Solid Tumors

NCT05985161
View directory record Official record
Conditions
Wilms Tumor, Rhabdoid Tumor, Malignant Peripheral Nerve Sheath Tumors, MPNST, Nephroblastoma, XPO1 Gene Mutation, Solid Tumor
Interventions
Selinexor
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
12 Months and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
16
States / cities
Los Angeles, California • Palo Alto, California • Washington D.C., District of Columbia + 13 more
Source: ClinicalTrials.gov public record
Updated May 6, 2026 · Synced Oct 4, 2026, 11:10 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 4, 2026, 11:10 AM EDT