Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-6
SponsorDarcy Krueger
About this trial
This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants.
This study is supported by research funding from the Office of Orphan Products Division (OOPD) of the US Food and Drug Administration (FDA).
Eligibility criteria
Qualifiers
0-6 months of age at the time of enrollment (subject must be <7 months of chronological age at time of randomization and treatment initiation). Corrected age must be at least 39 weeks (calculated by subtracting the number of weeks born before 40 weeks gestation from the chronological age).
Has a confirmed diagnosis of TSC based on established clinical or genetic criteria
Disqualifiers
Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG.
Has been treated in the past or is currently being treated at the time of enrollment with conventional anticonvulsant medications (AEDs), systemic (oral) mTOR inhibitors (such as rapamycin, sirolimus, or everolimus), ketogenic-related special diet, or another anti-seizure therapeutic agent, device, or procedure.
Has taken any other investigational drug as part of another research study, within 30 days prior to the baseline screening visit.
Has a significant illness or active infection at the time of the baseline screening visit
Trial design
Treatments tested in this trial
- Sirolimus
- Placebo
Treatment groups
64 Participants
are divided into 2 treatment groupsSponsors and collaborators
Darcy Krueger
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Sponsor institution
Source ClinicalTrials.gov