Clinical Trial: Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma

Brief Summary:

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.


Detailed Summary:

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).


Sponsor: Jonsson Comprehensive Cancer Center

Current Primary Outcome: Vestibular schwannoma volume [ Time Frame: 1 year (12 months) ]

Determine the effect of RAD001 on change in vestibular schwannoma volume (mm3) by MRI from baseline to 1 year.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Hearing [ Time Frame: 1 year (12 months) ]
    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) ]
    Determine the number of study subjects with adverse events by grade of severity


Original Secondary Outcome: Same as current

Information By: Jonsson Comprehensive Cancer Center

Dates:
Date Received: April 21, 2011
Date Started: July 1, 2015
Date Completion: February 1, 2019
Last Updated: April 25, 2017
Last Verified: April 2017