A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD
Status:
Completed
Trial end date:
2017-12-28
Target enrollment:
Participant gender:
Summary
The purpose of this study is to measure if the drug called Everolimus effects mTOR signaling
(an electrical activity signal in the brain) in patients with Tuberous Sclerosis Complex
(TSC) and Focal Cortical Dysplasia (FCD) with treatment resistant epilepsy (TRE) who will be
undergoing brain surgery. One group of patients will be treated with Everolimus, and another
will not. Researchers will determine if there is a difference in mTOR signaling between the
patients who were treated with Everolimus and those who were not. Previous studies have
suggested that Everolimus may reduce seizure activity in TSC patients by decreasing mTOR
signaling. Since patients with FCD may also have excess mTOR signaling brain activity,
Everolimus may also reduce seizure activity in these patients.
The drug Everolimus is approved by the Food and Drug Administration to treat specific types
of breast, pancreatic, and kidney cancer, a kidney tumor called an angiomyolipoma (common in
patients with TSC), and TSC patients who have a brain tumor called a subependymal giant cell
astrocytoma (SEGA). However, in this research it is considered to be an investigational since
it is not approved for reduction in mTOR signaling and a decrease in seizure frequency.
Researchers believe that Everolimus may be useful in reducing something called cortical
hyperexcitability, which is the excess brain activity that can contribute to seizures.
Phase:
Phase 2
Details
Lead Sponsor:
New York University School of Medicine NYU Langone Health