NCT05504291 · Children's Oncology Group
A Study to Give Treatment Inside the Eye to Treat Retinoblastoma
What this study is about
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).
View original scientific description
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.
Interventions
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
DRUG
Melphalan
Given I-VITRE
PROCEDURE
Ultrasound Biomicroscopy
Undergo UBM during EUA
DRUG
Vincristine
Given IV
Primary outcome measures
Feasibility success rate of intravitreal melphalan injection in combination with systemic chemotherapy
Time frame: Up to cycle 6 (1 cycle = 28 days)
A patient will be considered to have experienced intravitreal injection feasibility success if intravitreal melphalan can be delivered by cycle 6. If the treating physician does not inject because the eye has a full complete response (CR) for vitreous seeds after 2 cycles of systemic chemotherapy, it will be counted as a success in the feasibility analysis. Any feasibility evaluable patient who does not experience feasibility success will be considered a feasibility failure. For a bilateral patient with two Group D eyes with vitreous seeds, he/she will be categorized based on the worse results with the intent of being conservative, i.e., if intravitreal melphalan can be delivered in one eye but not the other by cycle 6 for any reason other than a CR of vitreal seeds, the patient will be deemed as experiencing a failure.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Patient must be \< 18 years of age at enrollment
- Patient must have newly diagnosed intraocular (localized) retinoblastoma and meet one of the following criteria:
- Unilateral Group D retinoblastoma with vitreous seeding; OR
- Bilateral retinoblastoma with worst eye Group D, with vitreous seeding present and the contralateral eye is Group A-C; OR
- Bilateral Group D retinoblastoma with at least one eye with vitreous seeding; OR
- Bilateral retinoblastoma with one Group D eye with vitreous seeding and one Group E eye where the Group E eye has been enucleated prior to any therapy. Note
Exclusion criteria
- for high-risk features
- Bilateral retinoblastoma with one Group D eye with vitreous seeding and one Group E eye where the Group E eye has not been enucleated prior to any therapy at the discretion of the treating physician. Note exclusion for patients with evidence of metastatic or extra orbital spread
- Patients must have a performance status corresponding to
Where
- Birmingham, Alabama
- Los Angeles, California
- Palo Alto, California
- Aurora, Colorado
- Atlanta, Georgia
- Ann Arbor, Michigan
- St Louis, Missouri
- Durham, North Carolina
- Akron, Ohio
- Cleveland, Ohio
- Memphis, Tennessee
- Austin, Texas
And 4 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Feb 17, 2026 · Source of record for eligibility and locations