Summary

Eligibility
for people ages 16-65 (full criteria)
Location
at UCLA
Dates
study started
estimated completion
Principal Investigator
by Marco Giovannini, MD, PhD (ucla)

Description

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.

Official Title

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

Details

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).

Keywords

Neurofibromatosis Type 2 Neuroma, Acoustic Vestibular schwannoma Hearing Neurofibromatoses Neurofibromatosis 1 Neurofibroma Neurilemmoma Neuroma Neurofibromatosis 2 Everolimus RAD001, everolimus

Eligibility

You can join if…

Open to people ages 16-65

  • Diagnosis of NF2 by National Institutes of Health (NIH) criteria
  • Age ≥ 16 years
  • Progressive VS growth during the previous 12 months.
  • WHO performance status > or = 2
  • Adequate bone marrow, liver and renal function.
  • For women of childbearing potential, no pregnancy or breast-feeding
  • Willingness and ability to comply with scheduled visits, drug administration plan, laboratory tests, other study procedures, and study restrictions.
  • Willingness to provide informed consent

You CAN'T join if...

  • Inability to tolerate periodic MRI scans or gadolinium contrast.
  • Inability to tolerate periodic audiologic testing or to understand a language with established scoring for word recognition testing.
  • Inability to adequately perform volumetric measurement of at least 1 target lesionNote: Patients with cochlear or auditory brainstem implants may participate if a target lesion can be accurately assessed.
  • Radiation therapy for the target lesion in the 60 months preceding inclusion in the study.
  • Patients currently receiving anticancer therapies or who have received anticancer therapies within 4 weeks of the start of study drug.
  • Immunization with attenuated live vaccines within one week of study entry or during study period.
  • Presence of a fungal infection requiring systemic antifungal treatment at enrollment
  • Other malignancies within the past 3 years except for adequately treated carcinoma of the cervix or basal or squamous cell carcinomas of the skin.
  • Patients who have any severe and/or uncontrolled medical conditions.
  • Patients with a known hypersensitivity to everolimus or other types of rapamycin or to its excipients.
  • Patients unwilling to or unable to comply with the protocol

Location

  • University of California Los Angeles
    Los Angeles California 90095-7286 United States

Lead Scientist at UC Health

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Jonsson Comprehensive Cancer Center
ID
NCT01345136
Phase
Phase 2
Study Type
Interventional
Last Updated