New pill aims to ease fatigue and brain fog in rare mitochondrial disease
NCT ID NCT06402123
First seen Jun 25, 2026 · Last updated Jul 08, 2026 · Updated 3 times
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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43 people
The number who actually took part.
- Started
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Sep 2024
- Finished
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Jun 2026
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 75 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Signed consent form. 2. 18 to 75 years of age. 3. Diagnosed with MELAS based on the presence of each of the following criteria: 1. A documented pathogenic variant in a mitochondrial DNA (mtDNA) gene. 2. History of one or more stroke-like episodes (SLEs) with magnetic resonance imaging (MRI) findings consistent with stroke-like lesions. 4. Scores below normal average on the iDSST and GMLT. 5. Reports fatigue due to MELAS. 6. Can complete at least 1 sit-to-stand in the 30-second test interval. 7. Completes all at-home weekly activities independently during the Screening Period (caregiver may help set up device/app, log-in, etc.). 8. Other criteria per the protocol. Exclusion Criteria: 1. Systolic blood pressure (BP) 90 mmHg or diastolic BP 60 mmHg. 2. Orthostatic hypotension when measured after standing from a semi-recumbent/supine position. 3. Active cancer significant enough to confound the results of this study. 4. Severe gastrointestinal dysmotility that may impact participation. 5. Recent history (within last 6 months) of platelet dysfunction, hemophilia, von Willebrand disease, coagulation disorder, other bleeding diathesis condition(s), or significant, nontraumatic bleeding episodes. 6. History of spontaneous fracture(s) that in the investigator's opinion represents a safety risk for trial participation. 7. Current use of prohibited medication (reviewed by investigator). 8. Any medical or other condition that the investigator thinks would preclude study participation. 9. Other exclusion criteria per protocol.
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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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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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