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Trial intelligence / Profile preview
Sirolimus in Leigh Syndrome
NCT06843811Phase 2Enrolling by invitation
- Start
- Feb 2025
- Primary completion
- Dec 2027
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label trial of sirolimus in Leigh syndrome (2 arms). Primary endpoints: Rate of Adverse Events at 2.5 years, Rate of Intercurrent Infection and Hospitalization at 2.5 years and 2 more. Started Feb 2025; status: enrolling by invitation.
02
Drugs
sirolimus
03
Conditions
Leigh syndrome
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