New hope for MELAS: experimental drug tested for Long-Term safety
NCT ID NCT06961344
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing the long-term safety of a daily oral drug called zagociguat in 44 adults with MELAS, a rare genetic disease that affects energy production in cells. All participants previously completed a lead-in study of the same drug. Researchers will monitor side effects over several years, with clinic visits every three to six months.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
zagociguat (15 mg oral tablet once daily)
What this could lead to
If successful, this study could show that zagociguat is safe enough for long-term use in MELAS, potentially leading to a treatment that helps manage this rare mitochondrial disease.
What could go wrong
This is a small, open-label extension study with no placebo group, so results are preliminary. It only includes people who already tolerated the drug in a prior trial, which may not reflect broader patient experiences.
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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Fondazione Policlinico Universitario Agostino Gemelli
Rome, 00168, Italy
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LMU Klinikum, Friedrich-Baur-Institute, Department of Neurology
Munich, 80336, Germany
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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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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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Royal Melbourne Hospital
Melbourne, Victoria, 3052, Australia
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The Newcastle upon Tyne NHS Foundation Trust
Newcastle upon Tyne, NE14LP, United Kingdom
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UC San Diego - Altman Clinical and Translational Research Institute
La Jolla, California, 92037, United States
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UT Health Houston
Houston, Texas, 77030, United States
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University College London Hospital. The National Hospital for Neurology and Neurosurgery, Centre for Neuromuscular Diseases
London, WC1N 3BG, United Kingdom
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