Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
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 Administr
| Condition(s) | Tuberous Sclerosis Complex, Epilepsy |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | 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). |
| Who can participate | Inclusion Criteria: 1. 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). 2. Has a confirmed diagnosis of TSC based on established clinical or genetic criteria Exclusion Criteria: 1. Prior history of seizures (clinical or electrographic) at the time of enrollment or identified on baseline EEG. 2. 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 a |
| Ages | 1 Day to 6 Months |
| Sex | All |
| Lead sponsor | Darcy Krueger |
| Locations | Birmingham, Alabama, United States; Los Angeles, California, United States; Palo Alto, California, United States; Aurora, Colorado, United States; Chicago, Illinois, United States; Boston, Massachusetts, United States (+5 more sites) |
| Start date | 2021-10-13 |
| NCT ID | NCT05104983 |
| Official listing | https://clinicaltrials.gov/study/NCT05104983 |