Can a new oral drug help tame leigh syndrome?
Disease control
Not yet recruiting
This trial tests an experimental oral drug, TTI-0102, in people aged 5 to 55 with Leigh syndrome spectrum, a rare genetic disorder that damages the brain and nerves. The study aims to find the right dose and check safety over 12 weeks of twice-daily treatment. Participants will h…
Phase: PHASE2 • Sponsor: Thiogenesis Therapeutics, Inc. • Aim: Disease control
Last updated Jul 26, 2026 00:00 UTC