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Clinical trial

A Pilot Phase 2 Study With Long-Term Extension to Assess the Safety and Efficacy of Sirolimus in Patients With Leigh Syndrome Caused by Genetically-Confirmed Mitochondrial Respiratory Chain Deficiency

NCT06843811 is registered in ClinicalTrials.gov with Matthew Demczko as lead sponsor.

NCT NCT06843811
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Registry record

As filed in ClinicalTrials.gov.

Title
A Pilot Phase 2 Study With Long-Term Extension to Assess the Safety and Efficacy of Sirolimus in Patients With Leigh Syndrome Caused by Genetically-Confirmed Mitochondrial Respiratory Chain Deficiency
Lead sponsor
Matthew Demczko
Phase
Phase 2
Overall status
Enrolling by invitation
Start date
February 27, 2025
Completion date
December 31, 2027
Enrollment
15 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

leigh syndrome