Charcot-Marie-Tooth disease: who works on it

One card per institution. Filter by target, by country, or show only institutions in EU-13 (Horizon Europe widening) countries.

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Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Withdrawn

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

  NCT03520751· Nationwide Children's Hospital· Updated 29 Jul 2026

  “Study is withdrawing to the retirement of the PI.”Registry text, quoted unchanged. Source

• Withdrawn

  Confirmatory Study of Govorestat in CMT-SORD

  NCT07191912· Applied Therapeutics, Inc.· Updated 29 May 2026

  “The study was withdrawn prior to enrollment as the sponsor decided not to proceed with study initiation.”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

• The Myelin Disorders Biorepository Project

  NCT03047369· Children's Hospital of Philadelphia· Source

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

  NCT01203085· University of Iowa· Source

• Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

  NCT01193088· University of Iowa· Source

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

  NCT01193075· Michael Shy· Source

• Muscle MRI Fat Fraction Before Surgery to Predict Strength Recovery After Foot-Drop Surgery in Charcot-Marie-Tooth Disease

  NCT07894536· Peking University Third Hospital· Source

• Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)

  NCT07447557· Elpida Therapeutics SPC· Source

• A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J

  NCT06151600· Elpida Therapeutics SPC· Source

• Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases

  NCT06666816· IRCCS Eugenio Medea· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

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