LEIGH SYNDROME

Clinical trials for LEIGH SYNDROME explained in plain language.

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Not yet finished but already full! 1 Terminated 1

Could a common drug slow a rare brain disease?

Disease control ENROLLING_BY_INVITATION

This study tests the drug sirolimus in 15 people with Leigh syndrome, a rare genetic disorder that damages the brain and muscles. The goal is to see if sirolimus is safe and can help manage symptoms. Participants must be between 6 months and 55 years old and have a confirmed gene…

Matched conditions: LEIGH SYNDROME

Phase: PHASE2 • Sponsor: Matthew Demczko • Aim: Disease control

Last updated May 18, 2026 12:03 UTC

LEIGH SYNDROME

Get alerted when new LEIGH SYNDROME trials appear

Could a common drug slow a rare brain disease?