A Phase II Study of RAD001 in the Treatment of Patients With Plexiform Neurofibromas (PN) Associated With Neurofibromatosis Type 1 (NF1)
1. Clinically definite diagnosis of NF1 according to the NIH consensus conference
2. Patients must have PN that have the potential to cause significant morbidity, such as
lesions that could compromise the airway or the great vessels, lesions that could
cause nerve compression, lesions that could result in major deformity or significant
3. Measurable disease: patient must have at least one measurable PN amenable to
volumetric MRI analysis.
1. Chronic treatment with systemic steroids or another immunosuppressive agent.
2. Evidence of an active optic glioma, malignant glioma, malignant peripheral nerve
sheath tumor, or other cancer requiring treatment with chemotherapy or radiation
3. Clinical evidence of significantly impaired lung function
4. Pregnancy or breast feeding.
5. Prior therapy with mTOR inhibitors (e.g.sirolimus, temsirolimus, everolimus).
6. No contraindications for MRI assessments
Other protocol-defined inclusion/exclusion criteria may apply