Clinical trialUNITERAREApr 3
New Recruiting Trial: A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)
Researchers are testing a new medicine called glycerol tributyrate to see if it can help people with two rare mitochondrial diseases: MELAS (a condition that causes brain inflammation, acid buildup in the blood, and stroke-like episodes) and LHON-Plus (a condition that damages the optic nerve and causes vision loss). This is an early-stage study that will enroll patients starting in April 2026.
WHY IT MATTERSThis is the first clinical trial testing glycerol tributyrate specifically in MELAS and LHON-Plus patients, offering a potential new treatment option for these currently untreatable mitochondrial disorders.