New drug trial offers hope for rare MELAS fatigue and brain fog

NCT ID NCT06402123

First seen Nov 01, 2025 · Last updated Apr 30, 2026 · Updated 24 times

Summary

This study tests an experimental drug called zagociguat in 43 adults with MELAS, a rare genetic disease that causes strokes, fatigue, and thinking problems. Participants take either 15 mg, 30 mg, or a placebo daily for 12 weeks. The goal is to see if the drug reduces fatigue and improves memory and thinking, while checking for side effects.

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Contacts and locations

Locations

  • Akron Children's Hospital

    Akron, Ohio, 44308, United States

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Columbia University Irving Medical Center

    New York, New York, 10027, United States

  • Fondazione Policlinico Universitario Agostino Gemelli

    Roma, 00168, Italy

  • Ludwig-Maximilians-University of Munich

    Munich, 80336, Germany

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • McMaster University Medical Center

    Hamilton, Ontario, L8N 3Z5, Canada

  • Mount Sinai - Ichan School of Medicine

    New York, New York, 10029, United States

  • Neurologic Institute Carlo Besta of Milan

    Milan, 20133, Italy

  • Neuroscience Research Australia

    Sydney, New South Wales, NSW 2031, Australia

  • Newcastle University

    Newcastle upon Tyne, NE14LP, United Kingdom

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • Royal Melbourne Hospital

    Melbourne, Victoria, 3052, Australia

  • Shared Health/University of Manitoba

    Winnipeg, Manitoba, R3A1R9, Canada

  • UC San Diego - Altman Clinical and Translational Research Institute

    La Jolla, California, 92037, United States

  • UCL Queen Square Institute of Neurology

    London, WC1N 3BG, United Kingdom

  • University Hospital Bonn

    Bonn, 53127, Germany

  • University of Texas Medical School at Houston

    Houston, Texas, 77030, United States

Conditions

Explore the condition pages connected to this study.