Clinical Trial: Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex
Brief Summary:
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.
Detailed Summary:
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
Sponsor: Children's Hospital Medical Center, Cincinnati
Current Primary Outcome: Number With Observed Adverse Side Effects [ Time Frame: During the entire study ]
Original Primary Outcome: 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.
Current Secondary Outcome: Overall Reduction in SEGA Tumor Volume. [ Time Frame: During the entire study ]
Original Secondary Outcome: Secondary Outcome Measures include overall reduction in SEGA tumor volume, neuropsychological testing, and change in cognitive function. These will be analysed after 5 subjects have completed 6 months on the study.
Information By: Children's Hospital Medical Center, Cincinnati
Dates:
Date Received: December 12, 2006
Date Started: January 2007
Date Completion:
Last Updated: October 17, 2014
Last Verified: October 2014
