Introduction

This family protocol summary is about the Children's Oncology Group study ACNS03333. 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.

ACNS0333 is a Phase III clinical trial. A trial is another word for a study. This study (clinical trial) is a therapeutic clinical trial. That means it is done to learn about treatment - its safety and how well it works. The purpose of a Phase III trial is to learn if a new treatment that is known to work in treating a type of cancer is better in some way than the standard treatment. For example, does it have better cure rates, longer control of disease, fewer or less serious side effects, or fewer days in the hospital?

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

ACNS0333

Official Title

Treatment of Atypical Teratoid/Rhabdoid Tumors (AT/RT) of the Central Nervous System with Surgery, Intensive Chemotherapy, and 3-D Conformal Radiation

Trial Opening Date

ACNS0333opened on December 8, 2008. The study has now enrolled 70 patients and is not currently enrolling patients. ACNS0333 might reopen if it's determined that additional patients are needed to answer the study questions.

General Patient Eligibility

· Age: < 22 years of age

· Diagnosis: Central Nervous System (CNS) AtypicalTeratoid/Rhabdoid Tumor (AT/RT)

· No prior radiation therapy or chemotherapy

Please consult your doctor to determine whether your child may participate in this study.

General Background and Study Goal

AT/RT is a rare and aggressive cancer that occurs in the brain and spinal cord and is found mostly in young children. AT/RT is the only childhood brain tumor that has been linked to a gene which might have a role in tumor development. AT/RT was first recognized as a separate tumor type in 1987 and ACNS0333 is the first cooperative group study of this disease.

The limited information from patients who have been treated for AT/RT shows that:

· there are certain intense chemotherapies that are active against AT/RT

• high-dose chemotherapy regimens that use stem cell rescue may be helpful
• a chemotherapy agent called methotrexate may be active against AT/RT
• giving radiation therapy early in treatment is important

There is no current standard treatment for children with AT/RT. This study will build on what has been learned already. It will test an experimental treatment plan that includes surgery, chemotherapy with stem cell rescue, and radiation therapy. The overall goal of this study is to find out what effects this treatment plan will have on children with AT/RT.

Other goals of this study are to:

· look at samples of tumor tissue and blood to see if there are any features of the tumor cells that can tell scientists more about the disease and better ways to treat AT/RT in the future

· develop a clinical and biologic database for future studies

Summary of the Treatment

The treatment plan involves surgery followed by chemotherapy plus stem cell rescue procedures. Induction chemotherapy will consist of two 21-day cycles during which stem cells will be collected (harvested). Consolidation chemotherapy will consist of three 28-day cycles. Each cycle of high dose consolidation chemotherapy is followed by an IV infusion of the patient's stem cells (called rescue).

Radiation therapy will be given to the location of the original tumor and in some cases may be given to the entire brain and spine if your doctor feels this is necessary. Radiation treatment is typically given 5 days a week for 6 weeks.

During the study, if the tumor gets larger the patient will be taken off study therapy and other treatment options will be discussed with the doctor.

Special Considerations

· In order to learn more about AT/RT tumor cells, COG scientists will study some of the tumor taken out in surgery and blood taken before treatment

· Consolidation therapy must occur at a COG-accredited transplant program

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:

Alyssa Reddy, MD

University of Alabama

Birmingham, AL 35233

FAMILY PROTOCOL SUMMARY REVIEW/APPROVALS