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OncoMatch/Clinical Trials/NCT05504291

A Study to Give Treatment Inside the Eye to Treat Retinoblastoma

Is NCT05504291 recruiting? Yes, currently enrolling (May 2026). This Phase 2 trial studies multiple treatments including Carboplatin and Etoposide for bilateral retinoblastoma.

Phase 2RecruitingChildren's Oncology GroupNCT05504291Data as of May 2026

Treatment: Carboplatin · Etoposide · Melphalan · VincristineThis 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.

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Extracted eligibility criteria

Disease stage

Required: Stage I

Performance status

ECOG 0–2(Ambulatory, capable of self-care)

Prior therapy

No prior treatment (treatment-naive required)
Max 0 prior lines

Cannot have received: systemic chemotherapy

Exception: cryotherapy and/or laser therapy (green or infrared) to the study eye(s) and non-study eye

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.

Cannot have received: intra-arterial chemotherapy

Exception: cryotherapy and/or laser therapy (green or infrared) to the study eye(s) and non-study eye

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.

Cannot have received: radioactive plaque

Exception: cryotherapy and/or laser therapy (green or infrared) to the study eye(s) and non-study eye

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.

Cannot have received: brachytherapy

Exception: cryotherapy and/or laser therapy (green or infrared) to the study eye(s) and non-study eye

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.

Cannot have received: radiation therapy

Exception: cryotherapy and/or laser therapy (green or infrared) to the study eye(s) and non-study eye

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.

Lab requirements

Blood counts

ANC >= 750/uL; Platelet count >= 75,000/uL (transfusion independent)

Kidney function

Serum creatinine based on age/sex (see table); OR 24-hour urine Creatinine clearance >= 70 mL/min/1.73 m^2; OR GFR >= 70 mL/min/1.73 m^2 by direct measurement; For patients < 1 month of age, serum creatinine < 1.5 x ULN or creatinine clearance or radioisotope GFR >= 70 mL/min/1.73 m^2

Liver function

Total bilirubin =< 1.5 x upper limit of normal (ULN) for age; SGPT (ALT) =< 135 U/L (ULN for SGPT (ALT) set to 45 U/L)

Peripheral absolute neutrophil count (ANC) >= 750/uL; Platelet count >= 75,000/uL (transfusion independent); Serum creatinine based on age/sex (see table); OR 24-hour urine Creatinine clearance >= 70 mL/min/1.73 m^2; OR GFR >= 70 mL/min/1.73 m^2 by direct measurement; Total bilirubin =< 1.5 x upper limit of normal (ULN) for age; SGPT (ALT) =< 135 U/L (ULN for SGPT (ALT) set to 45 U/L)

Structured fields extracted by AI. May contain errors — verify against the official protocol.

US trial sites

  • Children's Hospital of Alabama · Birmingham, Alabama
  • Children's Hospital Los Angeles · Los Angeles, California
  • Lucile Packard Children's Hospital Stanford University · Palo Alto, California
  • Children's Hospital Colorado · Aurora, Colorado
  • Children's Healthcare of Atlanta - Arthur M Blank Hospital · Atlanta, Georgia

Showing up to 5 US sites. See all sites on ClinicalTrials.gov →

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