Clinical Trials /

Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

NCT01345136

Description:

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.

Related Conditions:
  • Schwannoma
Recruiting Status:

Active, not recruiting

Phase:

Phase 2

Trial Eligibility

Document

Study of RAD001 for Treatment of <span class="go-doc-concept go-doc-biomarker">NF2</span>-related Vestibular Schwannoma

Title

  • Brief Title: Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma
  • Official Title: A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma
  • Clinical Trial IDs

    NCT ID: NCT01345136

    ORG ID: RAD001

    Trial Conditions

    Neurofibromatosis Type 2

    Neuroma, Acoustic

    Trial Interventions

    Drug Synonyms Arms
    RAD001, everolimus Everolimus, Afinitor RAD001 Treatment

    Trial Purpose

    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 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).

    Trial Arms

    Name Type Description Interventions
    RAD001 Treatment Experimental All subjects will be given RAD001 for 1 year (12 months). RAD001, everolimus

    Eligibility Criteria

    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

    Minimum Eligible Age: 16 Years

    Maximum Eligible Age: 65 Years

    Eligible Gender: Both

    Primary Outcome Measures

    Vestibular schwannoma volume

    Secondary Outcome Measures

    Hearing

    Number of adverse events

    Trial Keywords

    Neurofibromatosis Type 2

    Vestibular schwannoma

    Hearing