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)

WHY IT MATTERS

This 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.

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.

NCT ID: NCT06792500 Title: 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) Status: RECRUITING Phase: PHASE1, PHASE2 Sponsor: George Washington University Start date: 2026-04-01 URL: https://clinicaltrials.gov/study/NCT06792500 Source: UniteRare clinical trials database

YOU CAN ACT ON THIS

If you or a family member has MELAS or LHON-Plus, check the trial website (clinicaltrials.gov/study/NCT06792500) now to see if you meet eligibility criteria and contact the research team about enrollment.