Eye-Directed chemotherapy aims to save sight in children with retinoblastoma

NCT ID NCT01783535

First seen Jun 29, 2026 · Last updated Jun 30, 2026 · Updated 1 time

Summary

This phase 2 trial tests whether giving chemotherapy directly into the eye (subconjunctival carboplatin) along with systemic topotecan can shrink advanced retinoblastoma tumors in children with disease in both eyes. The goal is to preserve the eye and vision, avoiding removal. Participants also receive standard treatments like cryotherapy, laser therapy, or radiation as needed. The study includes children with newly diagnosed retinoblastoma and those whose disease spreads to the other eye after prior treatment.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
vincristine, topotecan, filgrastim, PEG-filgrastim, carboplatin
What this could lead to
If successful, this approach could help more children with advanced retinoblastoma keep their eye and vision, reducing the need for surgical removal.
What could go wrong
This is an early-phase study, so the treatment may not work for everyone. Chemotherapy carries risks like infection, low blood counts, and potential side effects from drugs given directly into the eye.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

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