A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma
This protocol is a Phase II, open-label, efficacy and safety study of single-agent RAD001 in
patients with NF2. During the study, subjects will receive continuous daily oral treatment
with RAD001 for up to 1 year or until tumor progression.
Primary Objective: To determine whether RAD001 has an effect on the VS growth in patients
with NF2 at a rate sufficient to submit the drug for further testing.
Secondary Objectives: To determine whether RAD001 has an effect on the volume of other
intracranial tumors, and to assess the effect of RAD001 on hearing function in patients with
NF2 (when applicable).
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Vestibular schwannoma volume
Determine the effect of RAD001 on change in vestibular schwannoma volume (mm3) by MRI from baseline to 1 year.
1 year (12 months)
Marco Giovannini, MD, PhD
House Research Institute
United States: Food and Drug Administration
|House Research Institute||Los Angeles, California 90057|