Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)

A Randomized, Double-blind, Placebo-controlled Study of Everolimus in the Treatment of Patients With Subependymal Giant Cell Astrocytomas (SEGA) Associated With Tuberous Sclerosis Complex (TSC)

This study evaluated the efficacy and safety of Everolimus in treating patients with Subependymal Giant Cell Astrocytomas associated with Tuberous Sclerosis Complex.

Inclusion Criteria:

• All Ages
• Definite diagnosis of Tuberous Sclerosis according to the modified Gomez criteria
• At least one Subependymal Giant Cell Astrocytoma of at least 1 cm in diameter
• Evidence of SEGA worsening as compared to prior MRI scans
• Females of child bearing potential must use birth control
• Written informed consent

Exclusion Criteria:

• SEGA related surgery is likely to be required in the opinion of the investigator
• Recent heart attack, cardiac related chest pain or stroke
• Severely impaired lung function
• Severe liver dysfunction
• Severe kidney dysfunction
• Pregnancy or breast feeding
• Current infection
• History of organ transplant
• Surgery within two months prior to study enrollment
• Prior therapy with a medication in the same class as Everolimus
• Uncontrolled high cholesterol
• Uncontrolled diabetes
• HIV
• Patients with metal implants thus prohibiting MRI evaluations

Other protocol-defined inclusion/exclusion criteria may apply