Mitochondrial encephalomyopathy – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Thu, 28 May 2026 11:31:05 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Mitochondrial encephalomyopathy – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 A study testing sonlicromanol in adults with genetically confirmed mitochondrial DNA 3243A>G variant causing mitochondrial disease https://clinicaltrials.eu/trial/a-study-testing-sonlicromanol-in-adults-with-genetically-confirmed-mitochondrial-dna-3243ag-variant-causing-mitochondrial-disease/ Wed, 29 Apr 2026 15:08:14 +0000 https://clinicaltrials.eu/trial/a-study-testing-sonlicromanol-in-adults-with-genetically-confirmed-mitochondrial-dna-3243ag-variant-causing-mitochondrial-disease/ This study involves people with Primary Mitochondrial Disease caused by a specific genetic change called the mitochondrial DNA 3243A>G variant. This genetic variant can lead to different forms of the disease, including MIDD (a form that can affect diabetes and hearing), MELAS (a condition that can cause stroke-like episodes and other symptoms), and conditions affecting multiple body systems. People with this genetic change often experience ongoing tiredness and muscle weakness that affects their daily activities. The study will test a medication called sonlicromanol, which is also known by its code name KH176, and compare it to a placebo to see if it helps improve symptoms.

The purpose of this study is to find out if sonlicromanol can help reduce physical tiredness and improve muscle strength and balance in adults with this genetic form of mitochondrial disease. The study will also look at whether the medication affects quality of life and overall well-being. Participants will take either sonlicromanol tablets or placebo tablets by mouth for 52 weeks. The maximum daily dose of the study medication is 180 milligrams.

During the study, participants will complete questionnaires on electronic devices to report how they are feeling, including questions about tiredness, daily activities, mood, and overall health. They will also perform a physical test called the 5 Times Sit-to-Stand Test, which measures how long it takes to stand up and sit down five times in a row. This test helps assess leg muscle strength and balance. Additional assessments will include heart examinations using standard echocardiography or cardiac MRI to check heart wall thickness. Participants will need to maintain their usual exercise routine throughout the study and attend regular appointments for safety checks and to monitor how well the treatment is working.

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