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

URL:

https://clinicaltrials.gov/show/NCT01345136

Trial Eligibility

Document

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

  • ORG STUDY ID: 13-001492
  • NCT ID: NCT01345136

Conditions

  • Neurofibromatosis Type 2
  • Neuroma, Acoustic

Interventions

DrugSynonymsArms
RAD001, everolimusEverolimus, AfinitorRAD001 Treatment

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

NameTypeDescriptionInterventions
RAD001 TreatmentExperimentalAll 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
Maximum Eligible Age:65 Years
Minimum Eligible Age:16 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Vestibular schwannoma volume
Time Frame:1 year (12 months)
Safety Issue:
Description:Determine the effect of RAD001 on change in vestibular schwannoma volume (mm3) by MRI from baseline to 1 year.

Secondary Outcome Measures

Measure:Hearing
Time Frame:1 year (12 months)
Safety Issue:
Description:Determine the effects of RAD001 treatment on hearing changes (from baseline to 1 year in the ear with the growing vestibular schwannoma.
Measure:Number of adverse events
Time Frame:1 year, 1 month (13 months)
Safety Issue:
Description:Determine the number of study subjects with adverse events by grade of severity

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Active, not recruiting
Lead Sponsor:Jonsson Comprehensive Cancer Center

Trial Keywords

  • Neurofibromatosis Type 2
  • Vestibular schwannoma
  • Hearing

Last Updated

March 12, 2021