Investigating sirolimus to prevent epilepsy in infants with Tuberous Sclerosis Complex
Sirolimus TSC Epilepsy Prevention Study (STEPS) IND#145820 11/8/2019
['FUNDING_R01'] · CINCINNATI CHILDRENS HOSP MED CTR · NIH-10482355
This study is looking at whether giving a medication called sirolimus to infants with Tuberous Sclerosis Complex can help prevent or delay seizures, with the hope of improving their quality of life.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CINCINNATI CHILDRENS HOSP MED CTR (nih funded) |
| Locations | 1 site (CINCINNATI, UNITED STATES) |
| Trial ID | NIH-10482355 on ClinicalTrials.gov |
What this research studies
This research focuses on infants diagnosed with Tuberous Sclerosis Complex (TSC), a genetic disorder that can lead to severe epilepsy and developmental issues. The study aims to determine if early treatment with sirolimus, a medication that inhibits a specific cellular pathway, can prevent or delay the onset of seizures in infants aged 0-12 months. By conducting a multicenter, randomized, double-blind, placebo-controlled clinical trial, researchers will assess both the effectiveness and safety of sirolimus in this vulnerable population. The goal is to provide a new treatment strategy that could significantly improve the quality of life for these infants.
Who could benefit from this research
Good fit: Ideal candidates for this research are infants aged 0-12 months who have been diagnosed with Tuberous Sclerosis Complex.
Not a fit: Patients who are older than 12 months or do not have a diagnosis of Tuberous Sclerosis Complex may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to a new preventative treatment for epilepsy in infants with TSC, potentially reducing the risk of lifelong developmental challenges.
How similar studies have performed: Previous research has shown promise in using mTORC1 inhibitors like sirolimus for treating various manifestations of TSC, indicating a potential for success in this novel application.
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.