Study on Treating Retinoblastoma with Eye Injections and Chemotherapy

Program Overview

This study is testing a new approach to treat retinoblastoma, a cancer of the eye, by adding a drug called melphalan directly into the eye along with standard chemotherapy. Retinoblastoma can be hereditary or occur randomly, and is more challenging to treat when tumors spread into the eye's jelly-like fluid, called vitreous seeds. Melphalan, along with other chemotherapy drugs like carboplatin, vincristine, and etoposide, may help kill cancer cells by damaging their DNA and stopping them from growing. The study hopes that adding melphalan early in treatment will improve outcomes for patients with these difficult-to-treat tumors.

Description

Primary Objective:

• To see if it's possible to safely give melphalan injections into the eye by the sixth cycle of treatment, along with standard chemotherapy drugs carboplatin, vincristine, and etoposide, for treating Group D retinoblastoma with vitreous seeds.

Secondary Objectives:

• To assess the safety and side effects of combining melphalan injections with standard chemotherapy for Group D retinoblastoma with vitreous seeds.
• To evaluate how effective this combination is in treating the cancer.

Exploratory Objectives:

• To check if eyes eligible for melphalan injections later in treatment could have been treated earlier by reviewing initial medical images.
• To standardize how samples from the eye are collected and stored for genetic testing.
• To explore the relationship between genetic changes in the cancer and treatment outcomes.
• To study the effects of melphalan on the eye tissue in cases where the eye is removed due to cancer progression.
• To evaluate the long-term vision potential in eyes treated with melphalan.

Treatment Plan:

• Cycles 1-2: Patients receive chemotherapy with carboplatin, vincristine, and etoposide every 28 days for up to 2 cycles, unless the cancer worsens or side effects are too severe. Eye exams and imaging are done under anesthesia at the start and before each cycle.

• Cycles 3+: Patients continue with the same chemotherapy and undergo eye exams to see if they can receive melphalan injections. If eligible, melphalan is injected into the eye once per cycle. Treatment continues every 28 days for up to 4 cycles, with possible additional melphalan injections.

Patients also have MRI scans and may provide eye fluid and tissue samples during the study. After treatment, follow-up visits occur at 4 weeks, then every 3 months for a year, and every 3-6 months for up to 5 years.

Eligibility Criteria