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
22 clinical trials for this condition and its sub-types.
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Broader categories
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Last chance access: vatiquinone for mitochondrial disease patients
Disease control NO_LONGER_AVAILABLEThis 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 ENROLLING_BY_INVITATIONThis 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: PHASE2 • Sponsor: Matthew Demczko • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC
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New study tests workplace coaching to keep Parkinson's patients on the job
Symptom relief OngoingThis study tests a personalized workplace intervention for 124 Dutch workers with Parkinson's disease, cerebellar ataxia, hereditary spastic paraparesis, or slowly progressive neuromuscular/mitochondrial disorders. A trained facilitator helps employees and their managers identify…
Phase: NA • Sponsor: Radboud University Medical Center • Aim: Symptom relief
Last updated Aug 16, 2026 00:00 UTC
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MRI scans could unlock secrets of rare muscle disease
Knowledge-focused ENROLLING_BY_INVITATIONThis study uses special MRI scans to measure how well muscles produce energy in people with mitochondrial disease. Researchers hope to learn more about the condition and develop a new tool to help diagnose and track it. The study involves 230 participants aged 7 to 75 with suspec…
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC
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Massive gene hunt launched for mysterious mitochondrial diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis 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