Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients

Launched by ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS · Dec 9, 2011

Keywords

ClinConnect Summary

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 4 years 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; to assess the effect of RAD001 on hearing function in patients with NF2 (when applica...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Diagnosis of NF2 by National Institutes of Health (NIH) criteria
• • Age ≥ 15 years
• • Progressive VS growth during the previous 12 months. Evidence of disease progression defined by progressive VS during the previous 12 months (\>20% increase in volume) in subjects who are at elevated risk for surgical complications (eg, deafness, lower cranial nerve injury, facial weakness) or who refuse surgery
• • 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.
• • Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent. Topical or inhaled corticosteroids are allowed.
• • 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