Clinical trial NCT01158651
Conditions: Glioma
Keywords: NF1 low-grade glioma progressive children NF Type 1 - low grade glioma - not responded to treatments
Sponsor: University of Alabama at Birmingham
Start date: 2010-07-10; Completion date: 2017-10-26
Enrollment: 23 participants; Allocation: NA; Intervention model: SINGLE_GROUP

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 in children with Neurofibromatosis type 1 (NF1). Additionally, the safety of RAD001 will be studied.

The study drug, RAD001, is a drug that may act directly on tumor cells by preventing tumor cell growth and development. RAD001 has been studied in participants with various types of cancer as a single agent (a drug that is used alone to treat the cancer) or in combination with a number of well known anticancer therapies. Information from these research studies suggests that RAD001 may help to shrink or slow the growth of low-grade gliomas.

In this research study, the investigators are looking to see the response of RAD001 in children with low-grade gliomas and NF1 that have either not responded to treatment or have come back after treatment. We are also looking for the highest dose of RAD001 that can be given safely in this patient population.

Detailed description:
After signing this consent form, you will be asked to undergo some screening tests or procedures to find out if you can be in the research study. These tests and procedures are likely to be part of regular glioma care and may be done even if it turns out that you do not take part in the research study. If you have had some of these tests or procedures recently, they may or may not have to be repeated.

A medical history, which involves questions about your health history, any medications you are taking or plan to take, and any allergies.

A physical exam, during which you will be asked about any problems that you might be having. Additionally, your clinician will check your vital signs (body temperature, heart rate, breathing rate, and blood pressure) and bodily systems (respiratory, nervous, digestive, etc). The doctor will also evaluate your performance status, which indicates how much your illness affects your activity level.

Blood tests, including tests to measure any effects of your disease. Approximately 1-2 teaspoons of blood will be drawn for these tests.

Urine test, which will be done to make sure your kidneys are functioning properly.

An assessment of your tumor using scans of the brain. MRI (Magnetic Resonance Imaging) will be used to look at and evaluate the tumor.

An Electrocardiogram (ECG), which measures the electrical activity of your heart

A pregnancy test for females of childbearing potential. A small amount of blood (about half a teaspoon) or urine will be drawn for this test.

If these tests show that you are eligible to participate in the research study, you will begin the study treatment. If you do not meet the eligibility criteria, you will not be able to participate in this research study.

Arms: arm 1: If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks).

You will take the study medication (tablets) by mouth, once a day during each course for as long as you are participating in this study. You will also be required to take an antibiotic during treatment to prevent infection.
Interventions: intervention 1: RAD001 (Everolimus)
Intervention descriptions: intervention 1: If you take part in this research study, you will be given a participant diary for each treatment course to help you keep track of when you take your RAD001. You will be required to bring the completed diary at each scheduled visit. A treatment "course" lasts 4 weeks and there will not be any breaks between courses. You may stay on study for a total of 12 courses (48 weeks).

You will take the study medication (tablets) by mouth, once a day during each course for as long as you are participating in this study. You will also be required to take an antibiotic during treatment to prevent infection.

Protocol documents: