Trial Information

A Phase II Trial of Proton Beam Radiation Therapy for Intra- and Periocular Retinoblastoma

External photon beam radiation therapy (which is current standard of care) has been used in
the treatment of retinoblastoma. It is a form of radiation therapy in which the radiation
is delivered by a machine pointed at the area to be radiated.

Proton beam radiation therapy is a form of external photon beam radiation therapy, but it
may be more effective because its adjusted dosing delivers less radiation to surrounding
areas of the tumor, which helps preserve other tissues and cause fewer side effects.

Before you can start treatment on this study, you will have what are called "screening
tests." These tests will help the doctor decide if you are eligible to take part in this
study. Your eye doctor will perform an assessment of your eye, including an exam while you
are under anesthesia. This exam will include a dilated eye exam, photography of your
affected eye (by a Ret-Cam), ultrasound of your affected eye, and imaging scans, such as
computed tomography (CT) or magnetic resonance imaging (MRI), if the doctor thinks it is
necessary. The assessments will be done to check and confirm the status of the disease.

After diagnosis of retinoblastoma, the study doctor will first see if external photon beam
radiation therapy is a reasonable treatment option for you. If external photon beam
radiotherapy is deemed appropriate for you, you will be eligible to receive proton beam
radiation therapy in this study.

If you are found to be eligible to take part in this study, you will be treated with proton
beam radiation therapy. Treatment will begin within 1 week of referral to the M. D. Anderson
Proton Center. It will be given daily for 5 days in a row each week (except for Saturdays,
Sundays, and holidays). The whole treatment will take about 4-6 weeks.

Within 2-4 weeks after completion of therapy, you will have an eye exam performed under
anesthesia (like the one during screening). You will have repeated eye exams under
anesthesia, depending on the appearance and response of the tumor to therapy. This will
occur about every 1-4 months.

You will continue to have these eye exams until the tumors are considered to be stable or
unless your disease gets worse.

If the disease gets worse or you experience any intolerable side effects, you may be taken
off treatment.

You will be required to have lifelong eye and medical assessments to continue to monitor you
for disease. If no disease is found 5 years after completion of proton beam radiation
therapy, you will be considered free of disease. You will then have annual (yearly) eye and
pediatric assessment through adulthood.

This is an investigational study. Up to 20 patients will take part in this study. All will
be enrolled at M.D. Anderson.