Leigh syndrome
MONDO:0009723A progressive neurological disease defined by specific neuropathological features associating brainstem and basal ganglia lesions.
Also known as: LS, LSS, Leigh disease, Leigh syndrome, Leigh syndrome spectrum, Leigh's disease, infantile subacute necrotizing encephalopathy, Leigh syndrome due to mitochondrial Complex 1 deficiency
21 clinical trials for this condition and its sub-types, 9 tagged with Leigh syndrome itself.
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Sub-types of Leigh syndrome
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Leigh syndrome with cardiomyopathy 0 trials
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Last chance access: vatiquinone for mitochondrial disease patients
Disease control Expanded access (ended)This program offered vatiquinone, an experimental liquid medication, to patients with inherited mitochondrial diseases like Leigh syndrome who had already completed a previous safety study. The goal was to continue treatment for those who might benefit, but enrollment is now clos…
Sponsor: Medical University of South Carolina • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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Could a common drug help kids with rare leigh syndrome?
Disease control By invitation onlyThis study tests the drug sirolimus (rapamycin) in 15 people aged 6 months to 55 years with genetically confirmed Leigh syndrome, a rare and serious mitochondrial disease. The goal is to see if the drug is safe and can help with symptoms like muscle weakness and developmental del…
Phase 2 • Sponsor: Matthew Demczko • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC
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Massive gene hunt launched for mysterious mitochondrial diseases
Knowledge-focused By invitation onlyThis study aims to discover new genetic mutations that cause mitochondrial disorders by analyzing tissue samples from up to 6,900 participants. It includes people with suspected or known mitochondrial diseases, such as MELAS or Leigh's Disease, who lack a genetic diagnosis. The r…
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC