NCT06843811View on ClinicalTrials.gov

Sirolimus for Leigh Syndrome

PHASE2Enrolling by invitationINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

February 27, 2025

Primary Completion Date

December 31, 2027

Study Completion Date

December 31, 2027

Conditions
Leigh Syndrome
Interventions
DRUG

Sirolimus

Sirolimus will be given at a starting dose of 0.8 to 1.3 mg/m2 twice daily, depending on subject age and weight.

Trial Locations (1)

19146

Children's Hospital of Philadelphia, Philadelphia

All Listed Sponsors
lead

Matthew Demczko

OTHER