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New drug trial aims to slow rare nerve disease progression

NCT ID NCT07191912

Summary

This study is testing whether a daily oral drug called govorestat can provide long-term benefits for people with a specific genetic form of Charcot-Marie-Tooth disease (CMT-SORD). About 155 patients aged 16-65 will take either the drug or a placebo for up to three years to see if it improves walking, strength, and daily function. The goal is to control the disease by lowering toxic sorbitol levels in the blood, which are thought to damage nerves.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • CHU La Timone

    Marseille, 13005, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Carlo Besta Neurological Institute

    Milan, 20133, Italy

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Charité - Universitätsmedizin Berlin

    Berlin, 10117, Germany

  • Clinic for Special Children

    Gordonville, Pennsylvania, 17529, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Institut de Myologie

    Paris, 75013, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Istanbul University

    Istanbul, 34093, Turkey (Türkiye)

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Koç University Hospital

    Istanbul, 34010, Turkey (Türkiye)

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • La Fe University and Polytechnic Hospital

    Valencia, 46026, Spain

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Sydney Childrens

    Sydney, 2013, Australia

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Uniklinik of the RWTH Aachen University

    Aachen, 52074, Germany

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University Medicine Gottingen (UMG)

    Göttingen, 37075, Germany

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • University of Iowa

    Iowa City, Iowa, 52242, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Vall d'Hebron Institut de Recerca (VHIR)

    Barcelona, 08035, Spain

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

Conditions

Explore the condition pages connected to this study.