RAD001

A Phase II Study of RAD001 (Everolimus) for Children with Neurofibromatosis Type 1and Chemotherapy-Refractory Progressive Low-Grade Gliomas

Goal

The RAD001 trial is designed to evaluate the effectives of the drug Everolimus in the treatment of patients with NF1 and low grade gliomas (brain tumors and optic gliomas) that have not responded to standard therapies.

Drug

Everolimus is a new medication that inhibits a protein called “mTOR,” which has been found to be hyperactive in some tumors associated with NF1.

Eligibility

The RAD001 trial will be open to patients with NF1 between the ages of 3 and 21 years who have low-grade gliomas (brain tumors or optic gliomas) and who have been treated with standard therapy (carboplatin in combination with other medications) but have not responded to this treatment. Patients must be physically and medically able to participate in the trial and have recovered from any side effects of prior treatment. A full list of criteria can be obtained by contacting one of the study sites.

Protocol

Participants will receive Everolimus as an oral medication once a day for up to one year of treatment. There will be regular clinical evaluations, imaging studies, and blood tests to monitor for side effects and effectiveness of the treatment. Further details about the protocol can be obtained by contacting one of the study sites.

Current Status

The RAD001 trial is expected to begin recruitment in early 2010. Up to 21 patients will be enrolled in the study.

Further Information

For further information, please contact one of the study sites or the Operations Center.