Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma
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. Secondary objectives include determining if RAD001 treatment will improve hearing ability in NF2 patients.
RAD001 is an oral drug that is approved by Food and Drug Administration (FDA) for other types of tumors, it is not approved by the FDA for treatment of NF2 related tumors.
Neurofibromatosis Type 2
Drug: RAD001, everolimus
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma|
- Vestibular schwannoma volume [ Time Frame: 1 year (12 months) ] [ Designated as safety issue: No ]Determine the effect of RAD001 on change in vestibular schwannoma volume (mm3) by MRI from baseline to 1 year.
- Hearing [ Time Frame: 1 year (12 months) ] [ Designated as safety issue: No ]Determine the effects of RAD001 treatment on hearing changes (from baseline to 1 year in the ear with the growing vestibular schwannoma.
- Number of adverse events [ Time Frame: 1 year, 1 month (13 months) ] [ Designated as safety issue: Yes ]Determine the number of study subjects with adverse events by grade of severity
|Study Start Date:||December 2012|
|Estimated Study Completion Date:||February 2017|
|Estimated Primary Completion Date:||February 2017 (Final data collection date for primary outcome measure)|
Experimental: RAD001 Treatment
All subjects will be given RAD001 for 1 year (12 months).
Drug: RAD001, everolimus
Adults: 10 mg p.o. daily dose, age 16 - 17: 3.0 mg/m2 p.o. daily
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).
Please refer to this study by its ClinicalTrials.gov identifier: NCT01345136
|United States, California|
|University of California Los Angeles|
|Los Angeles, California, United States, 90095-7286|
|Principal Investigator:||Marco Giovannini, MD, PhD||University of California, Los Angeles|