Sirolimus for Leigh Syndrome
At a glance
Conditions
NCT06843811
Where you'd take part
This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Children's Hospital of Philadelphia
Philadelphia, Pennsylvaniano site contact published
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
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.