Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

Sponsor

Jonsson Comprehensive Cancer Center (Other)

Overall Status

Active, not recruiting

CT.gov ID

NCT01345136

Collaborator

Novartis Pharmaceuticals (Industry)

Enrollment

Location

Arm

103.1

Anticipated Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Neurofibromatosis Type 2 Neuroma, Acoustic	Drug: RAD001, everolimus	Phase 2

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).

Study Design

Study Type:

Interventional

Actual Enrollment :

4 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (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

Actual Study Start Date :

Jul 1, 2015

Anticipated Primary Completion Date :

Feb 1, 2023

Anticipated Study Completion Date :

Feb 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
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

Arm

Intervention/Treatment

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

Outcome Measures

Primary Outcome Measures

Vestibular schwannoma volume [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 [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 [1 year, 1 month (13 months)]
Determine the number of study subjects with adverse events by grade of severity

Eligibility Criteria

Criteria

Ages Eligible for Study:

16 Years to 65 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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