New pill aims to ease fatigue and brain fog in rare mitochondrial disease
NCT ID NCT06402123
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 2b trial tests an oral drug called zagociguat in 43 adults with MELAS syndrome, a rare genetic disorder that causes fatigue, muscle weakness, and stroke-like episodes. Participants take either 15 mg, 30 mg, or a placebo daily for 12 weeks. The study measures changes in fatigue, cognitive performance, and leg strength, while monitoring for side effects.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
zagociguat (oral tablet)
What this could lead to
If successful, zagociguat could become a treatment to reduce fatigue and improve thinking and physical function in people with MELAS syndrome.
What could go wrong
This is a small, early Phase 2b trial with only 43 participants. The drug may not show clear benefits, and side effects are possible. Results may not apply to all MELAS patients.
Disclaimer
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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.
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.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Akron Children's Hospital
Akron, Ohio, 44308, United States
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Columbia University Irving Medical Center
New York, New York, 10027, United States
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Fondazione Policlinico Universitario Agostino Gemelli
Roma, 00168, Italy
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Ludwig-Maximilians-University of Munich
Munich, 80336, Germany
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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McMaster University Medical Center
Hamilton, Ontario, L8N 3Z5, Canada
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Mount Sinai - Ichan School of Medicine
New York, New York, 10029, United States
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Neurologic Institute Carlo Besta of Milan
Milan, 20133, Italy
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Neuroscience Research Australia
Sydney, New South Wales, NSW 2031, Australia
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Newcastle University
Newcastle upon Tyne, NE14LP, United Kingdom
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Royal Melbourne Hospital
Melbourne, Victoria, 3052, Australia
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Shared Health/University of Manitoba
Winnipeg, Manitoba, R3A1R9, Canada
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UC San Diego - Altman Clinical and Translational Research Institute
La Jolla, California, 92037, United States
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UCL Queen Square Institute of Neurology
London, WC1N 3BG, United Kingdom
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University Hospital Bonn
Bonn, 53127, Germany
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University of Texas Medical School at Houston
Houston, Texas, 77030, United States
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