Plain-English translation of NCT04681417 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
Retinoblastoma is a rare eye cancer that affects young children. This trial is testing two different approaches to treat it while trying to save the child's eyesight. Study 1 tests whether adding the drug topotecan to melphalan (a chemotherapy given directly into the eye's blood vessel) works better than melphalan alone. Study 2 tests a combination of three chemotherapy drugs (etoposide, carboplatin, and vincristine) given into the bloodstream, combined with local eye treatments. Both studies aim to shrink tumors and preserve vision without removing the eye.
Retinoblastoma has traditionally been treated by removing the affected eye, which is traumatic for children and families. This trial exists because doctors have developed new ways to deliver chemotherapy directly to the eye or in combinations that may control the cancer while letting children keep their sight.
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If your child is accepted into Study 1, they will receive chemotherapy injected directly into the blood vessel that feeds the eye, combined with local eye treatments, over several months. If accepted into Study 1, you'll have 2 to 6 cycles of intravenous chemotherapy given alongside eye treatments. In both studies, you'll attend regular clinic visits to monitor how the tumor is responding and how your child's vision is developing. The trial includes long-term follow-up to track both how well the cancer is controlled and whether your child's eyesight is preserved.
AI-generated summary from trial data · Oct 12, 2026 · Not medical advice
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