MELAS syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Wed, 24 Jun 2026 04:15:51 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png MELAS syndrome – 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.

]]> Study on the Safety and Effectiveness of Zagociguat for Treating MELAS in Patients https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-zagociguat-for-treating-melas-in-patients-2/ Wed, 29 Apr 2026 15:04:46 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-zagociguat-for-treating-melas-in-patients-2/ This clinical trial is focused on studying a condition called Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS). MELAS is a rare genetic disorder that affects the body’s ability to produce energy, leading to symptoms like muscle weakness, fatigue, and stroke-like episodes. The trial will evaluate the safety and effectiveness of an investigational drug called zagociguat, also known by its code name IW-6463. Zagociguat is taken in tablet form and is being tested to see if it can help improve fatigue and cognitive function, which includes mental processes like thinking and memory, in people with MELAS.

The purpose of this study is to assess how well zagociguat works and how safe it is for patients with MELAS. Participants in the study will receive either zagociguat or a placebo, which looks like the drug but does not contain the active ingredient. The study is designed as a crossover trial, meaning participants will receive both the drug and the placebo at different times during the study. This helps researchers compare the effects of the drug to the placebo. The study will last for about 12 weeks, during which participants will be monitored for any changes in their symptoms and any side effects they might experience.

Throughout the study, participants will undergo various assessments to measure their fatigue levels and cognitive abilities. These assessments include tests like the Groton Maze Learning Test (GMLT) and the International Digit Symbol Substitution Test (iDSST), which help evaluate cognitive function. The study will also track any adverse events, which are any unwanted effects that occur during the trial. By the end of the study, researchers hope to gather enough information to determine if zagociguat is a safe and effective treatment option for people with MELAS.

]]> Long-term safety study of zagociguat tablets in patients with MELAS syndrome (Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes) who completed previous trial https://clinicaltrials.eu/trial/study-on-the-safety-of-zagociguat-for-patients-with-melas-syndrome/ Wed, 29 Apr 2026 15:04:28 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-of-zagociguat-for-patients-with-melas-syndrome/ This study focuses on people with MELAS (Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes), which is a rare genetic condition that affects the body’s energy production in cells, leading to various symptoms including muscle weakness, neurological problems, and stroke-like episodes. The study will evaluate a medication called zagociguat, given as a 15 mg tablet taken by mouth.

The main purpose of this research is to assess the long-term safety of zagociguat in patients who have already completed a previous study of this medication. This is an open-label extension study, which means all participants will receive the active medication without use of a placebo.

The study will continue for approximately 156 weeks (3 years), during which participants will take the medication daily. Throughout the study, doctors will monitor participants’ health through various medical examinations and tests to ensure their safety and evaluate how well they tolerate the medication over an extended period.

]]> Study on the Effects of TTI-0102 for Patients with MELAS Syndrome https://clinicaltrials.eu/trial/study-on-the-effects-of-tti-0102-for-patients-with-melas-syndrome/ Wed, 29 Apr 2026 15:02:03 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-of-tti-0102-for-patients-with-melas-syndrome/ This clinical trial is focused on studying a rare condition known as Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS). This is a genetic disorder that affects the energy-producing structures in cells, leading to symptoms like muscle weakness, seizures, and stroke-like episodes. The study will test a new treatment called TTI-0102, which is an oral solution. TTI-0102 is a prodrug derivative, meaning it is a modified version of an existing medication called Cystagon®, which is used to treat similar conditions.

The purpose of the study is to evaluate how effective and safe TTI-0102 is for people with MELAS. Participants in the study will be randomly assigned to receive either TTI-0102 or a placebo, which is a substance with no active medication. The study will last for up to six months, during which participants will take the medication orally. Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. These check-ups will include tests to measure how well they can walk over a 12-minute period, as well as assessments of their overall quality of life and fatigue levels.

In addition to monitoring the main symptoms of MELAS, the study will also look at how the body processes TTI-0102 by measuring levels of certain substances in the blood, such as cysteamine and pantothenic acid (vitamin B5). This will help researchers understand how the drug works in the body. The study aims to provide valuable information on whether TTI-0102 can be a safe and effective treatment option for people living with MELAS.

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