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

• Status: Completed
• Trial end date: 2017-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Assistance Publique - Hôpitaux de Paris
• Treatments: Everolimus; Sirolimus

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