New hope for MELAS: experimental drug tested for Long-Term safety
NCT ID NCT06961344
First seen Jun 27, 2026 · Last updated Jul 08, 2026 · Updated 2 times
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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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