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

GOALS

The RAD001 trial is designed to evaluate the visual response and the effectiveness 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.

DRUGS

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

ELIGIBILITY

NF1 participants must be 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.

PROTOCOL

This phase II study will evaluate the response to everolimus in children with neurofibromatosis type-1 and chemotherapy-refractory radiographically progressive low-grade gliomas. A treatment course will consist of 4 weeks of therapy and may be repeated up to 12 total courses (48 weeks). This study will provide data to evaluate other drugs (e.g. carboplatin) in future combination regimens with everolimus.

The potential clinical benefit will be tumor control and improvement in disease related symptoms. If there is activity in the target population, a pilot combination of everolimus with carboplatin for children with NF1 and progressive symptomatic low-grade gliomas will be developed.

PROTOCOL CHAIRS

• Karen Wright, MD, Chair
• John Perentesis, MD, Co-Chair
• Alyssa Reddy, MD, Co-Chair
• Brian Weiss, MD, Co-Chair

CURRENT STATUS

The study is complete and closed. Please see trial results via publications or visit clinicaltrials.gov/ct2/show/NCT01158651.