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


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.


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


The RAD001 trial is open to patients with NF1 between the ages of 1 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.


Participants 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 has met its enrollment goals and is currently in the data analysis phase.

Further Information

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