Home › Trials › NCT05504291

Treatment for Retinoblastoma Using Eye Injections

Intravitreal Melphalan for Intraocular Retinoblastoma

Phase 2 Interventional Children's Oncology Group · NCT05504291

This study is testing if giving children with a specific type of eye cancer an injection of a drug directly into their eye, along with regular chemotherapy, can help treat their condition safely and effectively.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment26 (estimated)
AgesN/A to 18 Years
SexAll
SponsorChildren's Oncology Group Research network
Drugs / interventionschemotherapy, radiation
Locations20 sites (Birmingham, Alabama and 19 other locations)
Trial IDNCT05504291 on ClinicalTrials.gov

What this trial studies

This phase II trial evaluates the safety and effectiveness of injecting melphalan directly into the eye, combined with standard chemotherapy, for treating retinoblastoma, a type of eye cancer in children. The study focuses on patients with Group D retinoblastoma that has vitreous seeding, which complicates treatment. Participants will receive systemic chemotherapy drugs alongside the intravitreal melphalan to assess the feasibility and toxicity of this approach. The trial also aims to explore the relationship between genetic markers and treatment outcomes.

Who should consider this trial

Good fit: Ideal candidates are children under 18 with newly diagnosed intraocular retinoblastoma, specifically those with Group D retinoblastoma and vitreous seeding.

Not a fit: Patients with metastatic retinoblastoma or those who have already undergone enucleation of the affected eye may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve the effectiveness of retinoblastoma therapy and potentially reduce the risk of cancer recurrence.

How similar studies have performed: Other studies have explored similar approaches with localized treatments for retinoblastoma, indicating potential for success, though this specific method is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 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 Criteria:

* 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

Where this trial is running

Birmingham, Alabama and 19 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Bilateral RetinoblastomaChildhood Intraocular RetinoblastomaGroup D RetinoblastomaStage I RetinoblastomaUnilateral Retinoblastoma
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.