Tuberous Sclerosis Clinical Trial
Official title:
Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex
The purpose of the study is to evaluate the safety and potential side effects of everolimus
(an experimental drug) on a person with Tuberous Sclerosis Complex who also has been
diagnosed with a brain tumor (astrocytoma)
The hypothesis is that the drug will cause the tumor size to decrease, and may have
beneficial activity separate from effects on tumors in patients.
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
;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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