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ClinicalTrials.gov public records Last synced Oct 5, 2026, 2:26 PM EDT

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Showing 49–72 of 911 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neuromuscular; Disorder, Hereditary Clear all
Completed Phase 3 Interventional Results available

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy

NCT03703882
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne
Interventions
Edasalonexent, Placebo
Drug
Lead sponsor
Catabasis Pharmaceuticals
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
131 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
24
States / cities
Little Rock, Arkansas • Los Angeles, California • Sacramento, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jun 21, 2022 · Synced Oct 5, 2026, 2:26 PM EDT
Active, not recruiting No phase listed Observational

A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

NCT04475926
View directory record Official record
Conditions
Limb-girdle Muscular Dystrophy
Interventions
Not listed
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years and older
Enrollment
205 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
11
States / cities
Phoenix, Arizona • Little Rock, Arkansas • La Jolla, California + 8 more
Source: ClinicalTrials.gov public record
Updated Nov 6, 2025 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Phase 2 Interventional Results available

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03167255
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
NS-065/NCNP-01
Drug
Lead sponsor
NS Pharma, Inc.
Industry
Eligibility
4 Years to 10 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2021
U.S. locations
5
States / cities
Sacramento, California • Chicago, Illinois • St Louis, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 28, 2022 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Phase 2 Interventional

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

NCT05291091
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
Sevasemten 10 mg, Sevasemten 5 mg, Sevasemten 12.5 mg, Placebo
Drug
Lead sponsor
Edgewise Therapeutics, Inc.
Industry
Eligibility
12 Years to 50 Years · Male only
Enrollment
244 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
26
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 5, 2026, 2:26 PM EDT
Recruiting Phase 1 Interventional

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort

NCT04626674
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
2 Years and older · Male only
Enrollment
83 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
12
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 9 more
Source: ClinicalTrials.gov public record
Updated Sep 11, 2026 · Synced Oct 5, 2026, 2:26 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Observational Study of Digital Biomarkers of Myotonia and Gait in Adults and Children With Myotonic Dystrophy

NCT06089018
View directory record Official record
Conditions
Myotonic Dystrophy 1
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
12 Years to 90 Years
Enrollment
125 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
1
States / cities
Palo Alto, California
Source: ClinicalTrials.gov public record
Updated Oct 18, 2023 · Synced Oct 5, 2026, 2:26 PM EDT
Active, not recruiting Phase 1 Interventional

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

NCT06436742
View directory record Official record
Conditions
Congenital Myasthenic Syndrome, CMS
Interventions
ARGX-119, Placebo
Biological · Other
Lead sponsor
argenx
Industry
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
2
States / cities
Sacramento, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 24, 2026 · Synced Oct 5, 2026, 2:26 PM EDT
Recruiting Phase 4 Interventional Accepts healthy volunteers

Ultrasound Therapy In Cardiac Amyloidosis

NCT04667494
View directory record Official record
Conditions
Amyloidosis Cardiac
Interventions
Sonotherapy
Combination Product
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years to 90 Years
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 21, 2026 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Early Phase 1 Interventional

Tadalafil and Sildenafil for Duchenne Muscular Dystrophy

NCT01359670
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Tadalafil, Sildenafil
Drug
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
7 Years to 15 Years · Male only
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jan 14, 2020 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Phase 2 Interventional Results available

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

NCT02023866
View directory record Official record
Conditions
Inherited Mitochondrial Disease, Including Leigh Syndrome
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 5, 2026, 2:26 PM EDT
Enrolling by invitation Phase 3 Interventional

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

NCT07008469
View directory record Official record
Conditions
Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1, Myotonic Disorders, Steinert Myotonic Dystrophy, Steinert Disease
Interventions
Del-desiran (AOC 1001)
Drug
Lead sponsor
Avidity Biosciences, Inc.
Industry
Eligibility
16 Years and older
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
19
States / cities
Los Angeles, California • Stanford, California • Denver, Colorado + 16 more
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 5, 2026, 2:26 PM EDT
Terminated Not applicable Interventional Accepts healthy volunteers

Mendelian Reverse Cholesterol Transport Study

NCT01782027
View directory record Official record
Conditions
Cholesterol, HDL, Lipid Metabolism, Inborn Errors, Tangier Disease, LCAT Deficiency, Cholesteryl Ester Transfer Protein (CETP) Deficiency
Interventions
3H-cholesterol bound to albumin
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 75 Years
Enrollment
17 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Yogic Breathing Changes Salivary Components

NCT02108769
View directory record Official record
Conditions
Alzheimer Disease, Neurodegenerative Diseases, Cancer
Interventions
Yogic Breathing, Attention Control
Behavioral
Lead sponsor
Medical University of South Carolina
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013
U.S. locations
1
States / cities
Charleston, South Carolina
Source: ClinicalTrials.gov public record
Updated Apr 9, 2014 · Synced Oct 5, 2026, 2:26 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A

NCT03520751
View directory record Official record
Conditions
Charcot-Marie-Tooth Neuropathy Type 1A
Interventions
scAAV1.tMCK.NTF3
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
18 Years to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 5, 2026, 2:26 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Comparative Autoantibody and Immunologic Cell Marker Study

NCT02422875
View directory record Official record
Conditions
Autoimmune Diseases, Lupus Erythematosus, Systemic, Communicable Diseases
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
18 Years and older
Enrollment
2,500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2028
U.S. locations
3
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Not applicable Interventional

Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing

NCT03406208
View directory record Official record
Conditions
Neurofibromatoses, Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis
Interventions
Stress and Symptom Management Program 1, Stress and Symptom Management Program 2
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
228 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 24, 2025 · Synced Oct 5, 2026, 2:26 PM EDT
Suspended Phase 1 Interventional

A Phase 1a/1b Study to Evaluate the Safety of EZN-4176, in Adult Patients With Castration-Resistant Prostate Cancer

NCT01337518
View directory record Official record
Conditions
Prostatic Neoplasm
Interventions
EZN-4176
Drug
Lead sponsor
Enzon Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 18, 2012 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas

NCT01140360
View directory record Official record
Conditions
Neurofibromatosis, Neurofibromas
Interventions
Gleevec
Drug
Lead sponsor
Kent Robertson
Other
Eligibility
3 Years to 65 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 5, 2026, 2:26 PM EDT
No Longer Available No phase listed Expanded access

3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)

NCT02012933
View directory record Official record
Conditions
Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenia (CM)
Interventions
3,4-diaminopyridine
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
2 Years and older
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 11, 2019 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Phase 3 Interventional Results available

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

NCT05096221
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
delandistrogene moxeparvovec, placebo
Genetic
Lead sponsor
Sarepta Therapeutics, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Little Rock, Arkansas • La Jolla, California • Los Angeles, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jul 8, 2025 · Synced Oct 5, 2026, 2:26 PM EDT
Recruiting Not applicable Interventional

A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)

NCT06507748
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
AlgometRx Nociometer
Device
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 120 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Phase 2 Interventional Results available

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

NCT03038399
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day, Vamorolone 6.0 mg/day/day
Drug
Lead sponsor
ReveraGen BioPharma, Inc.
Industry
Eligibility
4 Years to 7 Years · Male only
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
6
States / cities
Davis, California • Gainesville, Florida • Orlando, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 20, 2021 · Synced Oct 5, 2026, 2:26 PM EDT
Completed Not applicable Interventional

Proprioception and Sensorimotor Control in Hereditary Sensory and Autonomic Neuropathy

NCT02876939
View directory record Official record
Conditions
Atunomic Neuropathy
Interventions
kinesiology tape around the hip, knee and ankle joints, No kinesiology tape around the hip, knee and ankle joints
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
16 Years to 40 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 22, 2020 · Synced Oct 5, 2026, 2:26 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)

NCT06792500
View directory record Official record
Conditions
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Interventions
Glycerol Tributyrate
Drug
Lead sponsor
George Washington University
Other
Eligibility
18 Years to 65 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2028
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 5, 2026, 2:26 PM EDT