A Study to Give Treatment Inside the Eye to Treat Retinoblastoma

Conditions

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

Eligibility Criteria

Sex

ALL

Age

N/A - 18 Years

Healthy Volunteers

Not accepted

Interventions

• Biospecimen Collection — PROCEDURE
  Undergo aqueous humor, tissue, and blood sample collection
• Carboplatin — DRUG
  Given IV
• Etoposide — DRUG
  Given IV
• Examination Under Anesthesia — PROCEDURE
  Undergo imaging of the eye during EUA
• Magnetic Resonance Imaging — PROCEDURE
  Undergo MRI
• Melphalan — DRUG
  Given I-VITRE
• Ultrasound Biomicroscopy — PROCEDURE
  Undergo UBM during EUA
• Vincristine — DRUG
  Given IV

Study Details

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.

Key Dates

Start date

Nov 4, 2022

Status verified

Feb 2026

Primary completion

Mar 31, 2028

Completion

Mar 31, 2028

Study Design

Enrollment

26 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Treatment (CVE, melphalan)
  See Detailed Description

Primary Outcome Measure

Feasibility success rate of intravitreal melphalan injection in combination with systemic chemotherapy [ Time Frame: Up to cycle 6 (1 cycle = 28 days) ]

Locations (17)

Find similar trials in Birmingham, AL

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