Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients (AFINF2)
The purpose of the study is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Single Arm, Single Center, Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma|
- effect of RAD001 on the VS growth by MRI [ Time Frame: 1 year ] [ Designated as safety issue: No ]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
- Effect of RAD001 on the volume of other intracranial tumors (MRI) and on hearing function (audiogram) [ Time Frame: 1 year and 2 years after inclusion in the study ] [ Designated as safety issue: No ]To determine if RAD001 has an effect on the volume of other intracranial tumors, to assess efficacy of RAD001 on hearing function, to determine whether RAD001 modulates signaling pathways in tumors removed during the course of the study.
|Study Start Date:||January 2012|
|Estimated Study Completion Date:||March 2014|
|Estimated Primary Completion Date:||December 2013 (Final data collection date for primary outcome measure)|
Patient with Neurofibromatosis Type 2 and Vestibular Schwannoma treated with RAD001.
10 mg per os / day or 05mg per os / day with 12 month
Other Name: Afinitor / rapamycin
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.
|Hôpital Beaujon, 100 boulevard du Général Leclerc|
|Clichy, France, 92110|
|Principal Investigator:||Michel Kalamarides, Professor||Assistance Publique - Hôpitaux de Paris|