New hope for MELAS: Long-Term safety trial of zagociguat begins

NCT ID NCT06961344

First seen Nov 01, 2025 · Last updated May 02, 2026 · Updated 22 times

Summary

This study looks at the long-term safety of a daily tablet called zagociguat in people with MELAS, a rare genetic disease that affects energy production and can cause strokes. About 44 participants who finished a previous study will take the drug for an extended period, with clinic visits every 3 to 6 months. The goal is to monitor any side effects and see if the drug remains safe over time.

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for MITOCHONDRIAL ENCEPHALOPATHY, LACTIC ACIDOSIS AND STROKE-LIKE EPISODES (MELAS SYNDROME) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

  • LMU Klinikum, Friedrich-Baur-Institute, Department of Neurology

    Munich, 80336, Germany

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • 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

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • Royal Melbourne Hospital

    Melbourne, Victoria, 3052, Australia

  • The Newcastle upon Tyne NHS Foundation Trust

    Newcastle upon Tyne, NE14LP, United Kingdom

  • UC San Diego - Altman Clinical and Translational Research Institute

    La Jolla, California, 92037, United States

  • UT Health Houston

    Houston, Texas, 77030, United States

  • University College London Hospital. The National Hospital for Neurology and Neurosurgery, Centre for Neuromuscular Diseases

    London, WC1N 3BG, United Kingdom

Conditions

Explore the condition pages connected to this study.