Trial Information

Multicenter Phase II Study for International Intraocular Retinoblastoma Classification Groups B, C & D Tumors Treated With Carboplatin-Etoposide-Vincristine-Cyclosporine-Focal Therapy Multimodality Protocol (OCRN Multicenter RB 2003)

OBJECTIVES:

Primary

- Compare the efficacy of neoadjuvant high-dose carboplatin and etoposide, vincristine,
and cyclosporine (CSA) followed by ophthalmic focal therapy comprising cryotherapy
and/or laser therapy to historical world data of chemotherapy treatment without CSA, in
terms of increasing the proportion of eyes that remain relapse free and do not require
external beam radiotherapy and/or enucleation, in patients with newly diagnosed Group
B, C, or D bilateral intraocular retinoblastoma.

Secondary

- Determine the toxicity of this regimen in these patients.

OUTLINE: This is a multicenter study.

Patients receive high-dose carboplatin IV over 30 minutes on day 1; vincristine IV over 5
minutes and high-dose etoposide IV over 25 minutes on day 2; cyclosporine IV over 1 hour
before chemotherapy and then over 2 hours after chemotherapy on days 1 and 2, and filgrastim
(G-CSF) subcutaneously once daily beginning on day 3 and continuing until day 16 or until
blood counts recover. Treatment repeats every 21 days for a total of 3 courses for patients
with Group B disease and a total of 6 courses for patients with Group C or D disease.

Patients undergo eye examination under anesthesia (EUA) at initial staging and then before
each course of chemotherapy. Patients with small peripheral tumors in eyes without retinal
detachment undergo minimal focal therapy (mainly cryotherapy) during EUA at initial staging
and then after chemotherapy courses 1 and 2. At EUA after the third and subsequent courses
of chemotherapy, patients with tumors that have sufficiently reduced in size undergo
additional cryotherapy or laser therapy. After completion of chemotherapy, patients with any
suspicious, active, or reactivated tumor undergo additional cryotherapy and/or laser therapy
during EUA approximately every 4-8 weeks (or at longer intervals) for up to 5 years (as
needed).

After completion of study chemotherapy, patients are followed every 3 months for 2 years,
every 6 months for 2 years, and then annually for 1 year.

PROJECTED ACCRUAL: A total of 71 patients will be accrued for this study within 2.4 years.