Introduction

This family protocol summary provides a general overview of the Children's Oncology Group (COG) study ACNS1022. It tells who is eligible and gives basic information about the study. More details about the study are in the consent form and protocol. You can get these from your oncologist.

ACNS1022 is a Phase II clinical trial. A trial is another word for a study. This study (clinical trial) is a therapeutic clinical trial. The purpose of a Phase II trial is to learn if a new treatment works in treating a specific type of cancer and how it affects the body. That means it is done to learn more about how well treatments work and the side effects of the treatments.

In a Phase II trial, patients with a specific type of cancer receive a new treatment using a dose that was found to be safe in a Phase I trial. Patients are checked for side effects of the treatment and to see whether the cancer shrinks or goes away.

Phase II trials are offered to patients whose disease has not responded to other less experimental treatments or to patients whose disease doesn’t have a standard treatment. Or Phase II trials may be offered to patients with the type of disease that responded well in Phase I trials.

In this study, each child is assigned to a study arm by a method called randomization. This is a process like flipping a coin that assures that each child has a fair and equal chance of being assigned to any of the study arms. This makes sure the study arms can be compared fairly.

In most clinical trials, we will not know which treatment is better until all of the children taking part in the trial have completed treatment and have been followed for several years. If one of the treatments is found to be better or safer than the others while the trial is still on-going, the trial will be stopped.  All children still in the trial will be given the treatment with the best results when possible.

It is important to understand that participating in a clinical trial is entirely voluntary. The decision about whether or not to participate will not affect the care provided by the health care team in any way. You can find additional information about participation in clinical trials at www.childrensoncologygroup.org. Always discuss any questions that you may have with your health care team.

Study Number

ACNS1022

Official Title

A Phase II Randomized Trial of Lenalidomide in Pediatric Patients with Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas

Trial Opening Date

ACNS1022 opened on March 19, 2012. The study reached accrual goals and is closed to further patient entry as of Wednesday, January 18, 2017.

General Patient Eligibility

• Age:
• Diagnosis: Pilocytic astrocytoma or optic glioma that has returned, progressed or become resistant to conventional therapy
• Very small infants cannot participate; patients must have a BSA > 0.4 m2 Patients must have been treated with no more than 2 prior anti-cancer regimens, and at least one of the two regimens must have included the drug carboplatin.

General Background and Study Goal

New ways to treat patients with juvenile pilocytic astrocytoma or optic glioma that recur or do not respond to primary treatment are needed.

Lenalidomide is an immunomodulatory agent. This means that it may help the immune system fight the tumor. Lenalidomide may also be an angiogenesis inhibitor. Angiogenesis is the formation of new blood vessels. Angiogenesis plays an important role in tumor growth, because tumors need blood vessels to grow and spread. A class of drugs called angiogenesis inhibitors are designed to prevent the formation of new blood vessels, thereby stopping or slowing the growth or spread of tumors.

Lenalidomide has been safely used in phase I studies to treat children and teens with different types of cancer, including some with brain cancer.

All children and teens who are part of this study will be treated with the drug lenalidomide. Half of the patients will receive a low dose of the drug, and half of the patients will receive a higher dose of the drug. Doctors would like to treat more children and teens with brain cancers so they can get more information about how well lenalidomide works.

Summary of the Treatment

This is a randomized study looking at two different doses of the anti-cancer drug, lenalidomide. Half of the children in this study will get a low dose of lenalidomide (Regimen A) and half will get a higher dose (Regimen B).

• Lenalidomide is taken daily for 21 days followed by a 7 day rest period. This constitutes a 28 day cycle.
• The study continues for a total of 26 cycles
• Patients or their guardians must keep a diary to record each dose of lenalidomide taken and any side effects

Special Considerations

Because lenalidomide stops new blood vessels from forming, it can cause severe damage to an unborn baby if a pregnant mother is receiving this drug. For this reason, female patients old enough to become pregnant are not eligible for this study unless they commit to complete abstinence or have been using 2 methods of birth control for at least 28 days (4 weeks) prior to study enrolment. Sexually active females must also agree to remain on 2 methods of birth control during treatment and for at least 28 days after completing protocol therapy.

Lenalidomide can only be taken as an intact capsule, so patients must be able to swallow a capsule to be on this study.

Risks and Side Effects

Chemotherapy can cause side effects during and after treatment. All patients will be closely monitored for possible side effects of the medicines. All risks and side effects will be explained by your treatment team during the consent process. They can answer any questions that you may have about giving permission for your child to be in the clinical trial or other aspects of care. Please refer to the consent form for a detailed explanation of the side effects associated with the treatment on this study.

Contact Information

Your child's oncologist and nurses are the best source for further information.

Study Chair
Katherine Warren, MD
National Cancer Institute
Bethesda, MD

FAMILY PROTOCOL SUMMARY REVIEW/APPROVALS