Conditions: Glioma Low-grade Glioma Astrocytoma
Keywords: RAD001
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 23
Healthy volunteers: no

Brief summary:
The purpose of this research study is to learn if the study drug RAD001 can shrink or slow the growth of low-grade gliomas. Additionally, the safety of RAD001 will be studied. RAD001 is a drug that may act directly on tumor cells by inhibiting tumor cell growth and proliferation.

Detailed description:
OBJECTIVES:

Primary

* To determine the response of children with chemotherapy-refractory or progressive low-grade gliomas to everolimus. Secondary
* To evaluate pharmacogenetic polymorphisms of cytochrome P450 3A4 \& 3A5 alleles and P-glycoprotein/MDR for their influence on the metabolism of everolimus in this patient population.
* To evaluate the role of Apolipoprotein E genotypes as predictors for development of hyperlipidemia during therapy with everolimus.
* To assess preliminary correlations of response with changes in pharmacodynamic parameters including p70s6 kinase activity in peripheral blood mononuclear cells.
* To describe the toxicity of everolimus when administered to this patient population.
* To characterize the pharmacokinetic profile of everolimus when administered to this patient population.

STATISTICAL DESIGN:

This study used a one-stage design to evaluate response to everolimus. If at least 3 responders are observed in 20 evaluable patients, then everolimus will be considered promising. If the true response rate is 5% (null hypothesis), the chance of concluding the treatment is active is 0.08 (Type I error). If the true response rate is 25% (alternative hypothesis), the chance of concluding the treatment is active is 0.91 (power).

Arms:
arm 1: Patients rcvd oral everolimus 5.0 mg/m2/day for a 28-day treatment course up to a total of 12 courses (48 weeks) if a patient had stable disease except if toxicity was unacceptable. Two dose reductions were permitted (3.0 5.0 mg/m2/day and 2.0 mg/m2/day).

Interventions: intervention 1: everolimus
Intervention descriptions: intervention 1: None

Protocol document: