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

Phase 2
18 Years
Not Enrolling
Glioblastoma Multiforme

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Trial Information

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

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.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Surgery Group: Percentage Change From the Baseline in S6 Kinase Levels

Outcome Description:

In the Surgery Group, the primary efficacy assessment was inhibition of Mammalian target of rapamycin (mTOR) as defined as ≥75% S6 phosphorylation. The occurrence of S6 phosphorylation was determined by phosphor-S6 immunohistochemical staining. Tumor cells from the initial surgical resection and from the salvage resection were used for assessments.

Outcome Time Frame:

Baseline and Day 7-9 (during salvage surgery)

Safety Issue:


Principal Investigator

Novartis Pharmaceuticals

Investigator Role:

Study Director

Investigator Affiliation:

Novartis Pharmaceuticals


United States: Food and Drug Administration

Study ID:




Start Date:

August 2007

Completion Date:

August 2009

Related Keywords:

  • Glioblastoma Multiforme
  • Glioblastoma Multiforme, GBM, RAD001, RAD
  • Glioblastoma



Beth Israel Deaconess Medical Center Boston, Massachusetts  02215
Massachusetts General Hospital Boston, Massachusetts  02114-2617
Seattle Cancer Care Alliance Seattle, Washington  98109
Northwestern University Chicago, Illinois  60611
Dana Farber Cancer Institute Boston, Massachusetts  02115
UCLA Los Angeles, California  90095
University of Cincinnati Cincinnati, Ohio  45267-0502
Duke University - Preston Robert Tisch Brain Tumor Center Durham, North Carolina  27710