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

Phase 2
16 Years
65 Years
Open (Enrolling)
Neurofibromatosis Type 2, Neuroma, Acoustic

Thank you

Trial Information

A Single Arm, Monocenter 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, and to assess the effect of RAD001 on hearing function in patients with
NF2 (when applicable).

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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

- 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

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Vestibular schwannoma volume

Outcome Description:

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

Outcome Time Frame:

1 year (12 months)

Safety Issue:


Principal Investigator

Marco Giovannini, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

House Research Institute


United States: Food and Drug Administration

Study ID:




Start Date:

December 2012

Completion Date:

January 2015

Related Keywords:

  • Neurofibromatosis Type 2
  • Neuroma, Acoustic
  • Neurofibromatosis Type 2
  • Vestibular schwannoma
  • Hearing
  • Neurilemmoma
  • Neurofibromatoses
  • Neurofibromatosis 1
  • Osteitis Fibrosa Cystica
  • Neurofibromatosis 2
  • Neuroma
  • Neuroma, Acoustic



House Research Institute Los Angeles, California  90057