Back to search results

Study of Melphalan Injections for Retinoblastoma

This study is looking at a new way to treat retinoblastoma (RB), a type of eye cancer, in children. It combines standard chemotherapy (carboplatin, etoposide) given through a vein with a drug called melphalan, which is injected directly into the eye. This approach is for children with newly diagnosed RB, especially those with vitreous seeding (cancer cells in the jelly-like fluid of the eye), which can make the cancer harder to treat. The main goal is to see if giving melphalan injections into the eye along with chemotherapy is practical and safe. Researchers also want to understand how well this combination works and its side effects. This study aims to enroll 26 children up to 18 years old.

Study design
This is an interventional study with a planned enrollment of 26 participants. It is testing the feasibility, safety, and effectiveness of combining intravitreal melphalan with systemic chemotherapy.
What's involved
Participants will undergo biospecimen collection (aqueous humor, tissue, blood samples), receive carboplatin and etoposide intravenously, and have eye examinations under anesthesia (EUA) and MRIs.
Compensation
Not stated in the trial record.
Follow-up
The primary outcome for feasibility is measured up to cycle 6 (1 cycle = 28 days).

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05504291

A Study to Give Treatment Inside the Eye to Treat Retinoblastoma

Recruiting
PHASE2Up to 18InterventionalTreatment
Children's Oncology Group
~26 participants
Updated 2026-08-25 on ClinicalTrials.gov
What's tested:Biospecimen CollectionCarboplatinEtoposideExamination Under AnesthesiaMagnetic Resonance ImagingMelphalan

At a glance

Recruiting sites
17 of 20 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Feasibility success rate of intravitreal melphalan injection in combination with systemic chemotherapy
Measured over Up to cycle 6 (1 cycle = 28 days)

Conditions

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

Explore related trials

Retinoblastoma

Where it's being run

20 sites across 15 states
Texas4
California2
Ohio2
Alabama1
Colorado1
Georgia1
Michigan1
Missouri1

Study leadership

  • Rachana Shah · PRINCIPAL_INVESTIGATOR · Children's Oncology Group

Who to contact

Site Public Contact
[email protected]
205-638-9285
Email the study team

Opens a ready-to-send draft in your own email app — review before sending.

Do you actually qualify for this trial?

Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.

Check eligibility for this trial ~2 min · HIPAA-protected · delete anytime
Eligibility criteria

Inclusion

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 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 Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients =\<16 years of age
Peripheral absolute neutrophil count (ANC) \>= 750/uL (must be performed within 7 days prior to enrollment unless otherwise indicated)
Platelet count \>= 75,000/uL (transfusion independent) (must be performed within 7 days prior to enrollment)
A serum creatinine based on age/sex as follows (must be performed within 7 days prior to enrollment; must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment):
1 month to \< 6 months = 0.4 (male and female)
6 months to \< 1 year = 0.5 (male and female)
1 to \< 2 years = 0.6 (male and female)
2 to \< 6 years = 0.8 (male and female)
6 to \< 10 years = 1.0 (male and female)
10 to \< 13 years = 1.2 (male and female)
13 to \< 16 years = 1.5 (male) and 1.4 (female)
\>= 16 years = 1.7 (male) and 1.4 (female) OR - a 24-hour urine Creatinine clearance \>= 70 mL/min/1.73 m\^2 OR - a glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard)
Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility
For patients \< 1 month of age, serum creatinine levels must be \< 1.5 x the treating institution's creatinine upper limit of normal (ULN) for patients \< 1 month of age or the creatinine clearance or radioisotope GFR must be \>= 70 mL/min/1.73 m\^2
The threshold creatinine values were derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the Center for Disease Control (CDC)
Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (must be performed within 7 days prior to enrollment; must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L (must be performed within 7 days prior to enrollment; must be repeated prior to the start of protocol therapy if \> 7 days have elapsed from their most recent prior assessment)
Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L

Exclusion

Patients with evidence of metastatic or extra-orbital spread
Patients must not have an invasive infection at time of protocol entry
Patients must not have had any prior anti-cancer therapy other than cryotherapy and/or laser therapy (green or infrared) to the study eye(s) and non-study eye, including systemic chemotherapy, intra-arterial chemotherapy, radioactive plaque, brachytherapy, or radiation therapy.
Note: A study eye is defined as being Group D with vitreous seeding. Patients may have had enucleation of one eye as long as the remaining eye is Group D with vitreous seeds
Patients with bilateral disease who undergo enucleation of a Group E eye prior to initiation of therapy and show evidence of high-risk histopathology features in the enucleated eye. High-risk histopathology includes choroid involvement \>= 3 mm, post lamina optic nerve involvement, full thickness scleral invasion or optic nerve invasion to the cut end
Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
Lactating females who plan to breastfeed their infants
Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
All patients and/or their parents or legal guardians must sign a written informed consent
All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

What this trial measures

  • Feasibility success rate of intravitreal melphalan injection in combination with systemic chemotherapyUp 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.