Primary Outcome Measures:
- 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. [ Time Frame: During the entire study ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Secondary Outcome Measures include overall reduction in SEGA tumor volume. [ Time Frame: MRIs at baseline, month 3 and end of study (month 6), ] [ Designated as safety issue: No ]
Intervention Details:
Drug: Everolimus
The initial everolimus dosage will be 3 mg/m2/d taken daily or every other day, with titration to achieve a trough serum level of 10-15 ng/ml. Patients unable to tolerate levels in this range will have their doses held or reduced 25% with the goal of achieving trough serum levels of 5-10 ng/ml. If a trough serum level of 10-15 ng/ml is not achieved at a dosage of 3 mg/m2/d, then further dosage escalation by 25% will be undertaken as tolerated, up to 5-6.5 mg/m2/d.
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 astrocytoma