Sirolimus to Prevent Epilepsy in Infants with Tuberous Sclerosis Complex (TSC)
Official title: Sirolimus TSC Epilepsy Prevention Study (STEPS) IND#145820 11/8/2019
Funded at Cincinnati Childrens Hosp Med Ctr · NIH-11563810 · Data updated 2026-11-12
A groundbreaking study tests whether giving sirolimus early to babies with TSC can prevent or delay seizures.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cincinnati Childrens Hosp Med Ctr NIH-funded |
| Lab location | 1 site (Cincinnati, United States) |
| Project start | September 10, 2021 |
| Project end | June 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $605,876 |
| NIH project number | 3R01FD007275-03S1 |
| Project ID | NIH-11563810 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are infants aged 0-12 months diagnosed with tuberous sclerosis complex.
✕Less likely to help
Some infants may not respond to treatment or may experience adverse effects that limit benefit.
What this research studies
This study enrolls infants aged 0-12 months with tuberous sclerosis complex to see if starting sirolimus early can stop seizures from developing and support better neurodevelopment. It is a multicenter, randomized, double-blind, placebo-controlled trial designed to test safety and seizure outcomes over time. If successful, the approach could change how we treat epilepsy risk in newborns with TSC and potentially improve long-term development.
Why it matters
Potential benefit: If sirolimus proves safe and effective, many infants with TSC could experience fewer seizures and better developmental outcomes.
How similar studies have gone: Prior studies in animal models and early human trials suggest mTOR inhibitors may delay seizures when given early, but this Phase IIb trial seeks definitive evidence.
Where this research is happening
-
Cincinnati Childrens Hosp Med Ctr
Cincinnati, United States Active
Researchers
- Principal investigator: Krueger, Darcy — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Krueger, Darcy
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.