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ClinicalTrials.gov public records Last synced May 22, 2026, 2:49 AM EDT

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Showing 1–24 of 71 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Charcot Marie Tooth Disease (CMT) Clear all
Recruiting No phase listed Observational
View directory record Official record
Conditions
Peripheral Neuropathy, Neuro-Degenerative Disease, Neuromuscular Diseases
Interventions
Not listed
Lead sponsor
Elpida Therapeutics SPC
Industry
Eligibility
Not listed
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2032
U.S. locations
3
States / cities
San Francisco, California • Iowa City, Iowa • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced May 22, 2026, 2:49 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Multiple Sclerosis
Interventions
ofatumumab with PRF, ofatumumab with AI
Combination Product
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 55 Years
Enrollment
284 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
13
States / cities
Fullerton, California • Aurora, Colorado • Basalt, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2021 · Synced May 22, 2026, 2:49 AM EDT
Terminated Phase 2 Interventional Results available
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 May 22, 2026, 2:49 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
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 May 22, 2026, 2:49 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2099
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 3, 2025 · Synced May 22, 2026, 2:49 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
Home Ankle Strengthing Program, Standard of Care
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 19, 2019 · Synced May 22, 2026, 2:49 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
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 May 22, 2026, 2:49 AM EDT
Recruiting No phase listed Observational
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 May 12, 2025 · Synced May 22, 2026, 2:49 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication &#X7C; Blood or Tissue &#X7C; Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
23
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 22, 2025 · Synced May 22, 2026, 2:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Charcot Marie Tooth Disease (CMT), Hereditary Sensory and Motor Neuropathy, Nerve Compression Syndromes, Tooth Diseases, Congenital Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
411 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 13, 2017 · Synced May 22, 2026, 2:49 AM EDT
Not yet recruiting No phase listed Observational
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Peripheral Neuropathy, Chronic Inflammatory Demyelinating Polyneuropathy
Interventions
NT3 blood draw
Diagnostic Test
Lead sponsor
Zarife Sahenk
Other
Eligibility
7 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 23, 2025 · Synced May 22, 2026, 2:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Bladder Outlet Obstruction, Multiple Sclerosis, Cauda Equina Syndrome, Enlarged Prostate With Lower Urinary Tract Symptoms, Parkinson Disease, Lower Urinary Tract Symptoms, Detrusor Underactivity, Spinal Cord Injuries
Interventions
Intermittent self-catheterisation
Device
Lead sponsor
ConvaTec Inc.
Industry
Eligibility
18 Years to 100 Years · Male only
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
2
States / cities
West Orange, New Jersey • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 29, 2024 · Synced May 22, 2026, 2:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth Disease, Multiple Sclerosis
Interventions
Electrical impedance myography (EIM)
Device
Lead sponsor
Skulpt, Inc.
Industry
Eligibility
35 Years to 80 Years
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
6
States / cities
Phoenix, Arizona • Miami, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 10, 2016 · Synced May 22, 2026, 2:49 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Persistent Post-concussive Syndrome, Concussion Post Syndrome
Interventions
Hyperbaric oxygen at 1.5 ATA, Sham treatment
Drug
Lead sponsor
Essentia Health
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Fargo, North Dakota
Source: ClinicalTrials.gov public record
Updated Dec 23, 2025 · Synced May 22, 2026, 2:49 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
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 May 22, 2026, 2:49 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Parkinson Disease
Interventions
Lithium aspartate
Dietary Supplement
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
45 Years to 80 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Williamsville, New York
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced May 22, 2026, 2:49 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, SPG4, SPG5A, Spastic Paraplegia 4, Spastic Paraplegia 5A, Early Onset Hereditary Spastic Paraplegia, Neuromuscular Diseases, Spastic Paraplegia, Hereditary
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
11
States / cities
Miami, Florida • Iowa City, Iowa • Boston, Massachusetts + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced May 22, 2026, 2:49 AM EDT
Recruiting Not applicable Interventional
View directory record Official record
Conditions
Charcot Marie Tooth Disease (CMT)
Interventions
Ankle-foot Orthoses
Device
Lead sponsor
Bopha Chrea
Other
Eligibility
25 Years to 55 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
North Liberty, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 22, 2025 · Synced May 22, 2026, 2:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
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 May 22, 2026, 2:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
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 May 22, 2026, 2:49 AM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
7 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 19, 2020 · Synced May 22, 2026, 2:49 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD)
Interventions
VCA-894A
Drug
Lead sponsor
Vanda Pharmaceuticals
Industry
Eligibility
Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 2, 2025 · Synced May 22, 2026, 2:49 AM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
ACE-083, 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
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
16
States / cities
Orange, California • Aurora, Colorado • Gainesville, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2022 · Synced May 22, 2026, 2:49 AM EDT
Completed Phase 4 Interventional Results available
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 May 22, 2026, 2:49 AM EDT