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ClinicalTrials.gov public records Last synced Sep 19, 2026, 4:54 AM EDT

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Showing 1–24 of 72 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Charcot-Marie-Tooth Disease Clear all
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

NCT07226297
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease Type 2D
Interventions
nL-GARS1-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
13 Years and older · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Sep 19, 2026, 4:54 AM EDT
Terminated No phase listed Observational

A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)

NCT03810508
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathy IV
Interventions
Not listed
Lead sponsor
Neurogene Inc.
Industry
Eligibility
Not listed
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Iowa City, Iowa • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 30, 2022 · Synced Sep 19, 2026, 4:54 AM EDT
Terminated Phase 2 Interventional Results available

Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

NCT03943290
View directory record Official record
Conditions
Facioscapulohumeral Muscular Dystrophy, Charcot-Marie-Tooth Disease
Interventions
ACE-083
Drug
Lead sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Industry
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
19
States / cities
Orange, California • Sacramento, California • Aurora, Colorado + 16 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2022 · Synced Sep 19, 2026, 4:54 AM EDT
Completed Phase 1Phase 2 Interventional

Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease

NCT00541164
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Coenzyme Q10, Coenzyme Q10
Drug · Dietary Supplement
Lead sponsor
Memorial Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Johnstown, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 14, 2013 · Synced Sep 19, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)

NCT06669949
View directory record Official record
Conditions
Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Interventions
no intervention
Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
Not listed
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Sep 19, 2026, 4:54 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

The Effects of Fish Oil Supplementation on the Brain Health of Collegiate Football Athletes

NCT04796207
View directory record Official record
Conditions
Traumatic Brain Injury (TBI), Chronic Traumatic Encephalopathy (CTE)
Interventions
Fish Oil (DPA+EPA 2:1 ratio) Capsules, High Oleic Safflower Oil Capsules
Dietary Supplement
Lead sponsor
University of Arizona
Other
Eligibility
18 Years to 25 Years · Male only
Enrollment
38 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2020
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated May 8, 2025 · Synced Sep 19, 2026, 4:54 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Charcot-Marie-Tooth Disease (CMT) Biological Sample Collection for IPSC Generation and Biobanking

NCT06203093
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Healthy
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
5 Years and older
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 2, 2025 · Synced Sep 19, 2026, 4:54 AM EDT
Completed Phase 4 Interventional Results available

Strategies in Maintenance for Patients Receiving Long-term Therapy (S.I.M.P.L.E.) With MMX (Multi-Matrix System) Mesalamine for Ulcerative Colitis (UC)

NCT00446849
View directory record Official record
Conditions
Ulcerative Colitis
Interventions
MMX Mesalamine
Drug
Lead sponsor
Shire
Industry
Eligibility
18 Years and older
Enrollment
290 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
62
States / cities
Birmingham, Alabama • Jonesboro, Arkansas • Long Beach, California + 53 more
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Sep 19, 2026, 4:54 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

NCT04798378
View directory record Official record
Conditions
Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies, Arthrogryposis, Spinal Cord Injuries, Charcot-Marie-Tooth, Stroke, Weakness of Extremities as Sequela of Stroke, Weakness Due to Upper Motor Neuron Dysfunction, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, Arm Paralysis, Cerebral Palsy
Interventions
Neurosleeve
Device
Lead sponsor
Thomas Jefferson University
Other
Eligibility
4 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
2
States / cities
Wilmington, Delaware • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 8, 2025 · Synced Sep 19, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Natural History Study for Charcot Marie Tooth Disease

NCT05902351
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease Type 2, Charcot-Marie-Tooth Disease, Type 2C, Charcot-Marie-Tooth Disease Type 2A2B, Charcot-Marie-Tooth Disease Type 2B2, Charcot-Marie-Tooth Disease Type 2A1, Charcot-Marie-Tooth Disease Type 4B1, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease Type 2B1, Charcot-Marie-Tooth Disease Type 2U (Diagnosis), Charcot-Marie-Tooth Disease Type 4A, Charcot-Marie-Tooth Disease, Type 4A, Axonal Form, Charcot-Marie-Tooth Disease Type 2A2A, Charcot-Marie-Tooth Disease Type 2S (Disorder), Charcot-Marie-Tooth Disease and Deafness, Charcot-Marie-Tooth Disease Type 4B2, Charcot-Marie-Tooth Disease Type 4H, Charcot-Marie-Tooth Disease Type 1F, Charcot-Marie-Tooth Disease Type 4C, Charcot-Marie-Tooth Disease Type 4E, Charcot-Marie-Tooth Disease Type 1D, Charcot-Marie-Tooth Disease Type 2Q (Diagnosis), Charcot-Marie-Tooth Disease Type 2A2, Charcot-Marie-Tooth Disease Type 2N (Diagnosis), Charcot-Marie-Tooth Disease Type 2B5, Charcot-Marie-Tooth Disease Type 2D, Charcot-Marie-Tooth Disease Type 4D, Charcot-Marie-Tooth Disease Type 2K, Charcot-Marie-Tooth Disease Type 2L (Diagnosis), Charcot-Marie-Tooth Disease Type 2T, Charcot-Marie-Tooth Disease Type 2I, Charcot-Marie-Tooth Disease Type 2J, Charcot-Marie-Tooth Disease Type 2E, Charcot-Marie-Tooth Disease Type 2G, Charcot-Marie-Tooth Disease Type 1C, Charcot-Marie-Tooth Disease Type 2R, Charcot-Marie-Tooth Disease Type 2O (Diagnosis), Charcot-Marie-Tooth Disease Type 2M, Charcot-Marie-Tooth Disease Type 2P, Charcot-Marie-Tooth Disease Type 2Y, Charcot-Marie-Tooth Disease Type 4F (Diagnosis), Charcot-Marie-Tooth Disease Type 4B3, Charcot-Marie-Tooth Disease Type 2H, HNPP, X-Linked Charcot-Marie-Tooth Disease, Charcot Marie Tooth Disease (CMT)
Interventions
Not listed
Lead sponsor
Hereditary Neuropathy Foundation
Government
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 16, 2026 · Synced Sep 19, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Vascular Lab Resource (VLR) Biorepository

NCT05628948
View directory record Official record
Conditions
Cardiovascular Diseases, Metabolic Disease, Peripheral Artery Disease, Carotid Disease, Aneurysmal Disease, Venous Disease, Thrombosis, Lymphedema, Lipedema, Non-Atherosclerotic Chronic Arterial Occlusive Disease, Vasculitis, Fibromuscular Dysplasia, Arterial Dissection, May-Thurner Syndrome, Thoracic Outlet Syndrome, Vasospasm
Interventions
Not listed
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years and older
Enrollment
500 participants
Timeline
2019 – 2031
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 18, 2025 · Synced Sep 19, 2026, 4:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

NCT01193075
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Not listed
Lead sponsor
Michael Shy
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2026
U.S. locations
18
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 13 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2025 · Synced Sep 19, 2026, 4:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Validation of Biomarkers in Amyotrophic Lateral Sclerosis (ALS)

NCT00677768
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease, Primary Lateral Sclerosis, Nervous System Diseases, Hereditary Spastic Paraparesis
Interventions
No intervention
Other
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
30 Years to 80 Years
Enrollment
475 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2015
U.S. locations
29
States / cities
Phoenix, Arizona • Orange, California • Jacksonville, Florida + 24 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2016 · Synced Sep 19, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

The Biomarkers in the Hyperbaric Oxygen Brain Injury Treatment Trial (BioHOBIT)

NCT04855396
View directory record Official record
Conditions
Traumatic Brain Injury
Interventions
Not listed
Lead sponsor
University of Michigan
Other
Eligibility
16 Years to 65 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
10
States / cities
San Diego, California • West Palm Beach, Florida • Iowa City, Iowa + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 21, 2026 · Synced Sep 19, 2026, 4:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Plasma Neurofilament Light and Its Relationship With Omega-3 Status and Soccer Heading in Women Soccer Players

NCT05457023
View directory record Official record
Conditions
Brain Trauma
Interventions
Plasma Neurofilament Light (NF-L), Omega-3 Index, HeadCount Questionnaire
Other
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
18 Years to 25 Years · Female only
Enrollment
27 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2023
U.S. locations
2
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Nov 12, 2023 · Synced Sep 19, 2026, 4:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Development and Validation of a Disability Severity Index for CMT

NCT01455623
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
8 Years and older
Enrollment
291 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 25, 2014 · Synced Sep 19, 2026, 4:54 AM EDT
Active, not recruiting Phase 3 Interventional

Efficacy and Safety of Remibrutinib Compared to Teriflunomide in Participants With Relapsing Multiple Sclerosis (RMS) (REMODEL-1)

NCT05147220
View directory record Official record
Conditions
Relapsing Multiple Sclerosis
Interventions
Remibrutinib, Teriflunomide
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 55 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
52
States / cities
Phoenix, Arizona • Tucson, Arizona • Fort Smith, Arkansas + 47 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Sep 19, 2026, 4:54 AM EDT
Completed No phase listed Observational

Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

NCT01203085
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
Up to 21 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2025
U.S. locations
9
States / cities
Palo Alto, California • Hartford, Connecticut • Orlando, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2025 · Synced Sep 19, 2026, 4:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Inherited Retinal Degenerative Disease Registry

NCT02435940
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced Sep 19, 2026, 4:54 AM EDT
Completed No phase listed Observational

Disability Severity Scale (DSI) and Hereditary Motor and Sensory Neuropathy Overall Disability Scale (HMSN-R-ODS)

NCT02194010
View directory record Official record
Conditions
Charcot Marie Tooth Disease, Inherited Peripheral Neuropathy
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
10 Years and older
Enrollment
563 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
2
States / cities
Iowa City, Iowa • Rochester, New York
Source: ClinicalTrials.gov public record
Updated Feb 6, 2019 · Synced Sep 19, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)

NCT05354622
View directory record Official record
Conditions
Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle, Motor Neuron Disease, Movement Disorders
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Month to 30 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Sep 19, 2026, 4:54 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Observational Study of Neuromuscular Function in CMT Type 1&2 and Healthy Controls

NCT04980807
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
Not listed
Lead sponsor
NMD Pharma A/S
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 6, 2022 · Synced Sep 19, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Natural History Study of Patients with HPDL Mutations

NCT05848271
View directory record Official record
Conditions
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease, Neonatal Encephalopathy, Mutation, Genetic Disease
Interventions
Patient Registry, Dry blood spots sampling
Other
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 29, 2025 · Synced Sep 19, 2026, 4:54 AM EDT
Completed Not applicable Interventional

Driving Ability in Patients With CMT 1A

NCT02357355
View directory record Official record
Conditions
CMT 1A
Interventions
UIHC Driving Simulator
Device
Lead sponsor
Jerath U Nivedita
Other
Eligibility
16 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 9, 2017 · Synced Sep 19, 2026, 4:54 AM EDT