A Study to Give Treatment Inside the Eye to Treat Retinoblastoma

Summary

This phase II trial tests the safety and side effects of adding melphalan (by injecting it into the eye) to standard chemotherapy in early treatment of patients with retinoblastoma (RB). RB is a type of cancer that forms in the tissues of the retina (the light-sensitive layers of nerve tissue at the back of the eye). It may be hereditary or nonhereditary (sporadic). RB is considered harder to treat (higher risk) when there are vitreous seeds present. Vitreous seeds are RB tumors in the jelly-like fluid of the eye (called the vitreous humor). The term, risk, refers to the chance of the cancer not responding to treatment or coming back after treatment. Melphalan is in a class of medications called alkylating agents. It may kill cancer cells by damaging their deoxyribonucleic acid (DNA) and stopping them from dividing. Other chemotherapy drugs given during this trial include carboplatin, vincristine, and etoposide. Carboplatin is in a class of medications known as platinum-containing compounds. It works in a way similar to the anticancer drug cisplatin, but may be better tolerated than cisplatin. Carboplatin works by killing, stopping or slowing the growth of cancer cells. Vincristine is in a class of medications called vinca alkaloids. It works by stopping cancer cells from growing and dividing and may kill them. Etoposide is in a class of medications known as podophyllotoxin derivatives. It blocks a certain enzyme needed for cell division and DNA repair and may kill cancer cells. Adding melphalan to standard chemotherapy early in treatment may improve the ability to treat vitreous seeds and may be better than standard chemotherapy alone in treating retinoblastoma.

Conditions

• Bilateral Retinoblastoma
• Childhood Intraocular Retinoblastoma
• Group D Retinoblastoma
• Stage I Retinoblastoma
• Unilateral Retinoblastoma

Interventions

DRUG

Melphalan

Given I-VITRE

PROCEDURE

Ultrasound Biomicroscopy

Undergo UBM during EUA

DRUG

Vincristine

Given IV

PROCEDURE

Biospecimen Collection

Undergo aqueous humor, tissue, and blood sample collection

DRUG

Carboplatin

Given IV

DRUG

Etoposide

Given IV

PROCEDURE

Examination Under Anesthesia

Undergo imaging of the eye during EUA

PROCEDURE

Magnetic Resonance Imaging

Undergo MRI

Sponsors & Collaborators

• Children's Oncology Group

  lead NETWORK

Principal Investigators

• Rachana Shah · Children's Oncology Group

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Max Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2022-11-04

Primary Completion

2028-03-31

Completion

2028-03-31

FDA Drug

Yes

Countries

• United States
• Australia
• Canada

Study Locations