Introduction

This family protocol summary provides a general overview of the Children's Oncology Group (COG) study ARET12P1. It tells who is eligible and gives basic information about the study goals. More details about the study are in the consent form. You can get these from your oncologist.

ARET12P1 is a clinical trial. A trial is another word for a study. The purpose of this trial is to learn if a new treatment works in treating a specific type of cancer and how it affects the body. It is done to learn more about how well treatments work and the side effects of the treatments.

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

ARET12P1

Official Title

A Multi-institutional Feasibility Study of Intra-Arterial Chemotherapy Given in the Ophthalmic Artery of Children with Retinoblastoma

Study Opening Date

ARET12P1 opened on 4/1/2014. The ARET12P1 committee hopes to enroll 42 patients. It is expected that this study will remain open for approximately 3 years. The study temporarily closed to accrual on 10/24/2016.

General Patient Eligibility

• Age: 6 months or older
• Diagnosis: newly diagnosed patients with group D retinoblastoma in one eye (unilateral)

Patients must have had no other therapy except for local therapy with cryotherapy (cold) or laser (heat) at the time of eye exam under anesthesia (EUA)

General Background and Study Goal

Retinoblastoma is the most common primary eye cancer in childhood. Group D retinoblastoma means that the tumor in the retina has spread into the inner jelly- like part of the eye. The standard treatment for group D retinoblastoma in one eye (unilateral) is surgery to remove the entire affected eye. The surgery is called enucleation. Treatments such as radiation, laser therapy, cryotherapy and/or chemotherapy are often used to control the tumor and prevent the need for enucleation. However, these therapies can have many side effects, and frequently, despite these treatments, the eye still needs to be removed.

The purpose of the study is to see if patients with Group D retinoblastoma in one eye can be successfully treated without enucleation and with minimal side effects. Doctors have started using a newer method for delivering chemotherapy for retinoblastoma. This treatment puts chemotherapy directly into the blood vessel that supplies the affected eye. This technique is called intra-arterial (IA) injection. IA injection of chemotherapy has been used in other trials in the United States and is well tolerated by patients.

Doctors want to study how easy or hard it is to give this treatment to children with unilateral retinoblastoma who would otherwise have the eye removed. The overall goals of the study are to:

• Find out the effects, good or bad, of chemotherapy given directly into the blood supply of the eye of children with unilateral retinoblastoma
• Find out how IA chemotherapy affects vision over time
• Find out if intra-arterial IA chemotherapy gives children with unilateral retinoblastoma a lower chance of needing to remove the eye

Summary of the Treatment

The treatment on this study uses a chemotherapy drug called Melphalan given directly into an artery that supplies blood to the eye. Children will receive IA Melphalan every 28 days for 3 months. IA Melphalan is given while the child is under anesthesia.

Treatment with intra-arterial melphalan is performed by a specialized doctor called an interventional radiologist. The doctor will insert a small tube into an artery in groin area. This tube is threaded to the blood vessel that supplies the retina. The melphalan will be injected into the tube. The injection of melphalan takes about 30 minutes. The entire procedure takes about 2 hours.

Patients will have a detailed examination of the eyes under anesthesia (called an EUA) by the ophthalmologist during the week prior to each IA Melphalan injection. Local retinoblastoma treatment with cryotherapy (cold) and or laser (heat) may be given as needed by the ophthalmologist during each EUA.

Special Considerations

• This trial is offered only at a limited number of COG hospitals
• During EUA, pictures will be recorded with a camera called a RetCam. These pictures will be reviewed by COG ophthalmologists to find out if the cancer is responding to the treatment
• Complete blood count (CBC) and physical examinations are needed weekly during this trial
• Vision will be tested once a year for four years, starting 1 year after completion of Melphalan treatment. This vision test includes matching letters on a wall chart with letters on a testing board. This test will help the study determine how well children treated with intra-arterial Melphalan can see

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 sources for further information.

Study Chair

Murali Mohan Chintagumpala MD
Baylor College of Medicine

FAMILY PROTOCOL SUMMARY REVIEW/APPROVALS