Promising drug for rare nerve disease trial pulled before start

NCT ID NCT07191912

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study was designed to test whether the drug govorestat could improve symptoms of Charcot-Marie-Tooth disease caused by sorbitol dehydrogenase deficiency (CMT-SORD), a rare nerve condition. It planned to enroll people aged 16 to 65 and compare govorestat to a placebo over 36 months. However, the trial was withdrawn before any participants were enrolled, so no data were collected.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

govorestat (AT-007)

What this could lead to

If it works, this could point toward a treatment that slows or improves symptoms of CMT-SORD, a rare nerve disease.

What could go wrong

This trial was withdrawn before any participants enrolled, so no results are available. The drug is still in early testing, and its benefits and risks are not yet proven.

Disclaimer Read more

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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Conditions

The condition(s) this trial relates to.

neuronopathy, distal hereditary motor, autosomal recessive 8

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • CHU La Timone

    Marseille, 13005, France

  • Carlo Besta Neurological Institute

    Milan, 20133, Italy

  • Charité - Universitätsmedizin Berlin

    Berlin, 10117, Germany

  • Clinic for Special Children

    Gordonville, Pennsylvania, 17529, United States

  • Institut de Myologie

    Paris, 75013, France

  • Istanbul University

    Istanbul, 34093, Turkey (Türkiye)

  • Koç University Hospital

    Istanbul, 34010, Turkey (Türkiye)

  • La Fe University and Polytechnic Hospital

    Valencia, 46026, Spain

  • Sydney Childrens

    Sydney, 2013, Australia

  • Uniklinik of the RWTH Aachen University

    Aachen, 52074, Germany

  • University Medicine Gottingen (UMG)

    Göttingen, 37075, Germany

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • Vall d'Hebron Institut de Recerca (VHIR)

    Barcelona, 08035, Spain