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Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2016 by Jonsson Comprehensive Cancer Center
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Jonsson Comprehensive Cancer Center Identifier:
First received: April 21, 2011
Last updated: August 23, 2016
Last verified: August 2016

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.

Condition Intervention Phase
Neurofibromatosis Type 2
Neuroma, Acoustic
Drug: RAD001, everolimus
Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:

Further study details as provided by Jonsson Comprehensive Cancer Center:

Primary Outcome Measures:
  • 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.

Secondary Outcome Measures:
  • 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

Estimated Enrollment: 25
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)
Arms Assigned Interventions
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
Other Names:
  • Everolimus
  • Afinitor

Detailed Description:

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).


Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01345136

Contact: Marco Giovannini, MD, PhD
Contact: Roberta Leyvas (310) 825-8496

United States, California
University of California Los Angeles Recruiting
Los Angeles, California, United States, 90095-7286
Contact: Marco Giovannini, MD, PhD   
Contact: Roberta Leyvas    (310) 825-8496   
Sub-Investigator: Phioanh (Leia) Nghiemphu, MD         
Sponsors and Collaborators
Jonsson Comprehensive Cancer Center
Novartis Pharmaceuticals
Principal Investigator: Marco Giovannini, MD, PhD University of California, Los Angeles
  More Information

Responsible Party: Jonsson Comprehensive Cancer Center Identifier: NCT01345136     History of Changes
Other Study ID Numbers: RAD001
Study First Received: April 21, 2011
Last Updated: August 23, 2016

Keywords provided by Jonsson Comprehensive Cancer Center:
Neurofibromatosis Type 2
Vestibular schwannoma

Additional relevant MeSH terms:
Neurofibromatosis 2
Neuroma, Acoustic
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Peripheral Nervous System Diseases
Neuromuscular Diseases
Neuroendocrine Tumors
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Cranial Nerve Neoplasms
Neoplasms by Site
Vestibulocochlear Nerve Diseases
Retrocochlear Diseases
Ear Diseases
Otorhinolaryngologic Diseases
Otorhinolaryngologic Neoplasms processed this record on April 25, 2017