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Alkylating agents

Intraocular Chemotherapy for Retinoblastoma

Phase 2
Recruiting
Led By Rachel C Brennan
Research Sponsored by Children's Oncology Group
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial tests the safety of adding a drug (melphalan) to standard chemotherapy to treat retinoblastoma (a type of cancer of the eye). It may help treat harder-to-treat cases and improve the ability to treat vitreous seeds.

Who is the study for?
This trial is for children under 18 with newly diagnosed retinoblastoma, a cancer of the retina. It's specifically for those with certain types of tumor spread within the eye but no cancer outside it. Kids must have good performance status and adequate organ function, as measured by blood counts and liver/kidney tests.
What is being tested?
The study is testing if injecting melphalan into the eye, along with standard chemo drugs like carboplatin, vincristine, and etoposide can better treat retinoblastoma. The focus is on improving treatment for tumors that have spread to the vitreous humor (the jelly-like fluid inside the eye).
What are the potential side effects?
Possible side effects include damage to healthy cells in the eye leading to vision problems or loss, general chemotherapy side effects like nausea, hair loss, fatigue, increased risk of infections due to low blood cell counts, and potential kidney or liver issues.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Feasibility success rate of intravitreal melphalan injection in combination with systemic chemotherapy
Secondary study objectives
Event-free survival (EFS)
Percentage of patients with grade 3 or higher toxicities
Other study objectives
Highly-recurrent RB somatic copy number alterations (SCNAs) tumor fraction
Long-term visual potential
Proportion of enucleated eyes with particular histopathological characteristics that are known or suspected to affect prognosis adversely
+3 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment (CVE, melphalan)Experimental Treatment8 Interventions
See Detailed Description
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Carboplatin
2014
Completed Phase 3
~6120
Magnetic Resonance Imaging
2017
Completed Phase 3
~1180
Biospecimen Collection
2004
Completed Phase 3
~2030
Vincristine
2003
Completed Phase 4
~2970
Etoposide
2010
Completed Phase 3
~2960
Melphalan
2008
Completed Phase 3
~1500

Find a Location

Who is running the clinical trial?

Children's Oncology GroupLead Sponsor
460 Previous Clinical Trials
239,972 Total Patients Enrolled
8 Trials studying Retinoblastoma
932 Patients Enrolled for Retinoblastoma
Rachel C BrennanPrincipal InvestigatorChildren's Oncology Group
Rachana ShahPrincipal InvestigatorChildren's Oncology Group

Media Library

Melphalan (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT05504291 — Phase 2
~13 spots leftby Dec 2026