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

• Status: Completed
• Trial end date: 2014-10-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novartis Pharmaceuticals
• Treatments: Everolimus; Sirolimus

Criteria

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