NCT06843811
Sirolimus for Leigh Syndrome
Enrolling by Invitation
PHASE2Ages 6–55InterventionalTreatmentMatthew DemczkoInvestigator-initiated
~15 participants
Updated 2026-03-31 on ClinicalTrials.gov
What's tested:Sirolimus
At a glance
Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Rate of Adverse Events
Measured over Up to 2.5 years
+3 more outcomes measured
Conditions
Where it's being run
1 sites across 1 statesPennsylvania1
Study leadership
- Matthew Demczko, MD · PRINCIPAL_INVESTIGATOR · Children's Hospital of Philadelphia
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
What this trial measures
- Rate of Adverse EventsUp to 2.5 years
Adverse events will be measured according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE). Incidence of AEs will be reported.
- Rate of Intercurrent Infection and HospitalizationUp to 2.5 years
Each infection and hospitalization will be reported.
- Incidence of abnormal safety lab valuesUp to 2.5 years
Count of clinically significant changes from baseline in safety labs
- Sirolimus Trough LevelUp to 2.5 years
Measure of Sirolimus level in the blood. Sirolimus dosage will be adjusted as needed in order to maintain a sirolimus trough level within a range of 5 to 10 ng/mL. The number of out-of-range results will be reported.