Study of Everolimus (RAD001) in Patients With Recurrent Glioblastoma Multiforme (GBM)

A Phase II Trial of RAD001 in Patients With Recurrent Glioblastoma Multiforme

This study will define the safety and efficacy of Everolimus (RAD001) administered daily in patients with glioblastoma multiforme (GBM)

This was a multicenter, open label, randomized study of RAD001 dosed daily in patients with recurrent GBM. The study was conducted with 2 parallel groups of patients. Group 1 was designed to study the biological effects of RAD001 in patients scheduled to undergo salvage surgical resection, and Group 2 was to enroll patients who were not scheduled for surgery. Patients in Group 1 were randomly assigned to one of three pre-surgery treatment groups (0, 5 or 10 mg/day RAD001 for 7 days). All patients in Group 2 were to receive a fixed daily dose of 10 mg/day oral RAD001.

Inclusion Criteria:

• Age 18 years of age or older
• Histologically confirmed Glioblastoma Multiforme (GBM)
• Radiographic evidence of disease progression
• Patients must have evaluable contrast enhancing tumor
• Availability of paraffin blocks or unstained pathology slides for biomarker studies
• Karnofsky Performance Status of greater than or equal to 60%

Exclusion Criteria:

• Prior treatment with Mammalian target of rapamycin (mTOR) inhibitor
• History of another malignancy within 3 years
• Cardiac pacemaker
• Ferromagnetic metal implants other than those approves as safe for use in Magnetic resonance imaging (MRI) scanners
• Claustrophobia
• Obesity
• Unstable systemic diseases
• Elevated cholesterol or triglycerides
• Radiation therapy or cytotoxic chemotherapy <=4 weeks prior to study enrollment. Patient must have recovered from the toxic effects of a prior chemotherapy.
• Patients must be off all enzyme inducing anticonvulsants for at least 2 week before study enrollment can occur
• Need for increasing dose of steroids. Patients on a stable or tapering dose of steroids >=7 days were permitted.