A Single Arm, Single Center, 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; to assess the effect of RAD001 on hearing function in patients with NF2
(when applicable); to determine whether RAD001 modulates signaling pathways in intracranial
NF2 tumors removed during the course of the study.
All patients will be treated with RAD001 10 mg p.o. daily dose (5mg if 15-17 years old with
cutaneous surface area <1.5m2) for one year except in case of unacceptable toxicity, death,
or discontinuation from the study for any other reason.
At 12 months an extension for another one year of RAD001 treatment will be discussed in case
of response. All patients will have a follow-up visit (including MRI) scheduled at 12 months
after the last dose of the study drug.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
effect of RAD001 on the VS growth by MRI
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
Michel Kalamarides, Professor
Assistance Publique - Hôpitaux de Paris
France: Ministry of Health