Intravitreal High-Dose Topotecan Treatment for Refractory or Recurrent Retinoblastoma in Children Aged 1 to 12 Years

What this Trial Is About

Retinoblastoma is the most common eye cancer in infants and children worldwide, often complicated by persistent or returning vitreous seeding which challenges treatment success. This trial investigates a new approach using high-dose Topotecan, a drug that damages cancer cell DNA and promotes tumor cell death, to treat resistant or returning retinoblastoma. The study aims to assess how well this targeted treatment works and its safety, including effects on the immune system and vision preservation. Participants will receive an intravitreal injection of Topotecan at a dose of 100 micrograms, given on the first day of the first week. Treatment frequency will be personalized by eye doctors based on the tumor stage and response. Follow-up visits occur every four weeks where eye fluid samples are collected and electroretinograms are performed to monitor treatment effects and eye health. Treatment continues until the vitreous seeding disappears or stabilizes. Throughout the study, patients will be regularly checked with eye exams, laboratory tests, and vision assessments. Researchers will measure tumor response after 28 days and monitor for side effects, immune reactions, and visual function over 13 weeks. This prospective trial lasts up to three years with ongoing evaluations to understand the drugs impact on eye tumors and safety.

High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed