Introduction

This family protocol summary is intended to provide a general overview of the Children's Oncology Group study ARET0321. It will provide information about the children that are eligible for this study and basic information regarding the specifics of the study. The informed consent and protocol contain the full details of the study. Those documents are available to you from your oncologist.

ARET0321 is a Phase 3 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?

ARET0321 compares two treatments. These treatments are called study arms. Usually, one study arm is the standard or the best proven current treatment. The other study arm has some changes or additions. The new treatment is thought to be at least as good as the standard treatment. It is not known if the new treatment will be better than the standard treatment.

In a Phase 3 trial, 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 getting checked for several years. If one of the treatments is found to be better or safer than the others while the trial is still going on, 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

ARET0321

Official Title

A Trial of Intensive Multi Modality Therapy for Extra- Ocular Retinoblastoma

Study Opening Date

ARET0321 opened 2/4/2008. The ARET0321 committee plans to enroll 90 patients. It is expected that this study will remain open for six years.

Study Closing Date

Study ARET 0321 reached accrual goals and was closed to further patient entry as of 11/11/2014.

General Patient Eligibility

Age: up to 10 years of age
• Diagnosis of extra-ocular retinoblastoma (spread outside of the eye) that has not been treated with chemotherapy or radiotherapy.
• Prior treatment with chemotherapy or radiotherapy for intra-ocular retinoblastoma is permissible.

General Background and Study Goal

Researchers want to know if they can improve the cure rate for extra-ocular retinoblastoma by giving more intensive (stronger) therapy. The overall goal of this study is:

• To find out whether more intensive therapy will cure more patients than other therapies used in the past
• To see how well the more intensive therapy makes the tumor shrink or disappear.
• To see what side effects occur with this therapy.

Summary of the Treatment

The treatment plan involves cancer fighting medicine called chemotherapy. The treatment on this clinical trial takes about 6 to 8 months. Multiple chemotherapy medicines are given. This chemotherapy is given intravenously primarily as an inpatient. The treatment plan is determined by disease stage:

Patients on this study, stage 2 and 3 will receive:

• 4 cycles of Vincristine, Cisplatin, Cyclophosphamide and Etoposide
• Followed by external beam radiation

Patients on this study, stage 4 will receive:

• Induction with 4 cycles of Vincristine, Cisplatin, Cyclophosphamide and Etoposide
• During Induction peripheral stem cell harvest. Healthy, special blood cells will be filtered out (apheresis) of the subjects blood and saved for later use
• Consolidation with high dose chemotherapy and stem cell rescue
• Possibly external beam radiation

Risks and Side Effects

Chemotherapy and radiation therapy can cause short- and long-term side effects. All patients will be closely monitored to reduce the likelihood of negative side effects. All risks and side effects will be explained in detail by your healthcare team during the consent process. They can answer any questions that you may have regarding participation on the clinical trial or other aspects of care. Please refer to the consent document for a detailed explanation of the side effects associated with the treatment on this study.

Contact Information

Your child's oncologist is the best source for further information. Physicians with eligible patients should refer questions to the principal investigator:

Study Chair

Ira Dunkel, MD
Memorial Sloan- Kettering cancer Center

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