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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 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

  • 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

Bilateral RetinoblastomaChildhood Intraocular RetinoblastomaGroup D RetinoblastomaStage I RetinoblastomaUnilateral Retinoblastoma

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 Aug 25, 2026 · Source of record for eligibility and locations

📊
1 of 26 participants interested
4% interest

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A short prescreen based on this study's listed criteria. A coordinator confirms eligibility — this is not a medical assessment.

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Study locations

Choose your preferred location, or select flexible during enrollment.

RECRUITING

Birmingham

Alabama

Location available
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Los Angeles

California

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Palo Alto

California

Location available
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Aurora

Colorado

Location available
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Atlanta

Georgia

Location available
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Ann Arbor

Michigan

Location available
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St Louis

Missouri

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Durham

North Carolina

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Akron

Ohio

Location available
View Akron location page

And 8 more locations available.

Express your interest

Share your contact details and a study coordinator can follow up about screening.

Secure & Confidential

Your information is protected and will only be shared with the research team.

What participation can include

  • Study-related care provided by the research team
  • Close monitoring by medical professionals
  • Possible compensation for time and travel*
  • The option to withdraw at any time
  • Contributing to medical research that may help future patients

*Compensation varies by study. Confirm details with coordinator.

Typical next steps

  1. 1.Submit this form
  2. 2.Phone screening
  3. 3.In-person assessment if eligible
  4. 4.Begin participation

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Looking for Retinoblastoma Treatment in Birmingham?

Join others in Alabama exploring innovative treatment options through clinical research

Retinoblastoma Treatment Options in Birmingham, Alabama

If you're searching for Retinoblastoma treatment in Birmingham, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Birmingham, Los Angeles, Palo Alto and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Retinoblastoma. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Alabama
Now Enrolling
Up to 26 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Retinoblastoma?

Potential Benefits

  • ✓Access to new treatment approaches before public availability
  • ✓Close monitoring by experienced medical professionals
  • ✓Study-related care provided at no cost
  • ✓Contribute to medical research for Retinoblastoma

What to Expect

  • →Initial screening to determine eligibility
  • →Regular check-ups and monitoring visits
  • →Possible compensation for time and travel
  • →You can withdraw at any time

Frequently Asked Questions About This Retinoblastoma Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT05504291. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.