Preventing Epilepsy in Infants with Tuberous Sclerosis Complex
Sirolimus TSC Epilepsy Prevention Study (STEPS) IND#145820 11/8/2019
This project gives a medication called sirolimus to infants with Tuberous Sclerosis Complex to see if it can stop or delay seizures and improve their development.
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 ID | NIH-10656395 on NIH RePORTER |
What this research studies
Tuberous Sclerosis Complex (TSC) often causes severe epilepsy that is hard to treat and can lead to lifelong learning difficulties. We know that a medication called sirolimus has helped some people with TSC, but it might work even better if given very early. This project is a clinical trial where we will give sirolimus or a placebo to infants with TSC, aged 0-12 months, before they start having seizures. We want to find out if this early treatment can prevent or delay seizures and help with their brain development. We will also carefully check if the medication is safe for these young children.
Who could benefit from this research
Good fit: Ideal candidates are infants aged 0-12 months who have been diagnosed with Tuberous Sclerosis Complex but have not yet experienced seizures.
Not a fit: Patients who have already developed epilepsy or are older than 12 months may not be eligible for this specific early intervention approach.
Why it matters
Potential benefit: If successful, this medication could prevent or significantly delay the onset of epilepsy and improve brain development in infants with Tuberous Sclerosis Complex.
How similar studies have performed: Previous studies in mouse models and human trials suggest that early treatment with similar medications might be more effective against epilepsy and developmental issues in TSC.
Where this research is happening
Cincinnati, United States
- Cincinnati Childrens Hosp Med Ctr — Cincinnati, United States (Active)
Researchers
- Principal investigator: Krueger, Darcy — Cincinnati Childrens Hosp Med Ctr
- Study coordinator: Krueger, Darcy
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 below.