Charcot-Marie-Tooth disease trials in United States

In United States 10 Also abroad 32

10 trials · From ClinicalTrials.gov, checked 13 November 2026. Ask the study team whether a trial fits you.

No phaseTaking people
The Myelin Disorders Biorepository Project
Where
Akron, Atlanta, Baltimore, Boston, Charlottesville, Chicago, Columbus, Houston, Los Angeles, Minneapolis, Orange, Palo Alto, Philadelphia, Pittsburgh, Rochester, Sacramento, Salt Lake City, San Diego, San Francisco, Seattle, Washington D.C., Winston-Salem
People
12,000
Run by
Children's Hospital of Philadelphia
See details and how to joinNCT03047369
No phaseTaking people
Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2
Where
Ann Arbor, Aurora, Baltimore, Boston, Chapel Hill, Columbus, Hartford, Houston, Iowa City, Los Angeles, Maple Grove, Memphis, Miami, Palo Alto, Philadelphia, Rochester, Seattle
People
1,050 worldwide
Run by
University of Iowa
See details and how to joinNCT01193088
No phaseTaking people
Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others
Where
Ann Arbor, Aurora, Baltimore, Boston, Chapel Hill, Columbus, Hartford, Houston, Iowa City, Los Angeles, Memphis, Miami, Minneapolis, Palo Alto, Philadelphia, Rochester, Seattle
People
5,000 worldwide
Run by
Michael Shy
See details and how to joinNCT01193075
No phaseTaking people
Natural History Study for Charcot Marie Tooth Disease
Where
New York
People
10,000
Run by
Hereditary Neuropathy Foundation
See details and how to joinNCT05902351
Phase 1/2Taking people
Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)
Where
Iowa City, Palo Alto
Age
3 years to 20 years
People
8
Run by
Elpida Therapeutics SPC
See details and how to joinNCT07447557
No phaseTaking people
A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders
Where
Bethesda
Age
1 month to 100 years
People
999
Run by
National Human Genome Research Institute (NHGRI)
See details and how to joinNCT06092346
No phaseTaking people
Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Where
San Francisco
People
28
Run by
University of California, San Francisco
See details and how to joinNCT06669949
No phaseTaking people
Inherited Retinal Degenerative Disease Registry
Where
Columbia
People
20,000
Run by
Foundation Fighting Blindness
See details and how to joinNCT02435940
Phase 1/2Enrolling by invitation
Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)
Where
Houston
Age
13 years and over
People
1
Run by
n-Lorem Foundation
See details and how to joinNCT07226297
No phaseTaking people
CMT Gait, Mobility, Balance - AOFAS Grant
Where
North Liberty
Age
25 years to 55 years
People
66
Run by
Bopha Chrea
See details and how to joinNCT07188415