Experimental drug targets toxic sugar to halt rare nerve disease

NCT ID NCT05397665

Summary

This study is testing whether a drug called AT-007 can help control a rare, inherited nerve disease called SORD deficiency. The drug aims to lower high levels of a harmful sugar alcohol (sorbitol) in the body, which is thought to damage nerves and cause walking difficulties. About 56 adults with the condition will take either AT-007 or a placebo daily for up to two years to see if the drug improves their walking speed and other measures of function.

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Contacts and locations

Locations

  • Axon Clinical, s.r.o.

    Prague, Prague, 150 00, Czechia

  • Fondazione IRCCS Istituto Neurologico "Carlo Besta"

    Milan, Milano, 20133, Italy

  • Hassman Research Institute

    Berlin, New Jersey, 08009, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • University College of London

    London, London, WC1E 6BT, United Kingdom

  • University of Colorado

    Aurora, Colorado, 80045, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of Miami

    Coral Gables, Florida, 33146, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Rochester

    Rochester, New York, 14642, United States

Conditions

Explore the condition pages connected to this study.