Everolimus for Children With Recurrent or Progressive Ependymoma
Status:
Active, not recruiting
Trial end date:
2021-12-30
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate the anti-tumor activity of Everolimus among children
with recurrent or progressive ependymoma. Recurrent or progressive ependymoma is incurable
and has very limited treatment options. The rationale for this study is based upon both
pre-clinical and clinical considerations: Immunohistochemistry studies have demonstrated that
20 out of 23 (87%) pediatric ependymomas are immunoreactive for phosphorylated S6, a
biomarker that often predicts response to mTOR pathway-targeted therapy. Furthermore,
children with with multiply recurrent ependymomas have had objective and durable responses to
the mTOR inhibitor, Sirolimus (Rapamune, Pfizer). As a result of this pre-clinical and
clinical data, this study will further investigate the activity of an mTOR pathway inhibitor,
Everolimus, against children with recurrent or progressive ependymomas. In this study,
Everolimus will be administered at a dose and schedule that have previously been demonstrated
as safe and effective in children. Children may take Everolimus for up to 2 years on this
study, until tumor progression or unacceptable toxicity.