Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex
Tuberous Sclerosis Complex (TSC)is a genetic disorder with a birth incidence of
approximately one in six thousand. Five to twenty percent of patients with TSC will develop
astrocytoma, a slowly progressive tumor. They grow and cause damage to surrounding brain
tissue, blockage of spinal fluid (hydrocephalus), blindness, trouble walking, seizures, and
brain damage. If untreated, they can be fatal. Standard treatment involves surgery to
remove the tumor; however surgery may itself cause brain damage, bleeding, or infection, as
well as other complications. Studies have shown that everolimus suppresses the chemicals
that cause tumors to grow in tuberous sclerosis, and may cause them to shrink.
The primary objective of this study is to find out the effects of everolimus on astrocytomas
in a six month trial in patients with Tuberous Sclerosis who have been diagnosed with an
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary Outcome Measure will be incidence of reported and observed adverse side effects as a percentage of patients enrolled in the study and treated with everolimus. These will be analysed at 6 months after study initiation.
During the entire study
David N Franz, M.D.
Children's Hospital Medical Center, Cincinnati
United States: Food and Drug Administration
CCHMC IRB# 06-07-50
|Cincinnati Children's Hospital Medical Center||Cincinnati, Ohio 45229-3039|