Plain-English translation of NCT06792500 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This clinical trial is testing whether a medication called can help treat two rare inherited diseases caused by problems in mitochondria (the energy-producing parts of cells): MELAS, which causes stroke-like episodes and seizures, and LHON-Plus, which affects vision and nerve function. You would take the medication by mouth for up to 18 months while researchers monitor how well it works and how your body tolerates it.
Currently, there are very few treatment options for these severe mitochondrial diseases. This trial is exploring whether the medication can help reduce symptoms and improve quality of life by supporting how cells produce energy.
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You would start with a two-month baseline phase where researchers gather detailed medical information, perform blood tests, imaging scans (MRI), eye exams if needed, and assess your current symptoms and quality of life. Then you would enter a six-month dose-escalation phase where you gradually take increasing amounts of the medication by mouth to find the best dose for you. Finally, you would take your personalized dose for 12 months while researchers monitor your health, symptoms, and any side effects through regular visits and testing.
AI-generated summary from trial data · Jun 1, 2026 · Not medical advice
United States