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

Launched by EYE & ENT HOSPITAL OF FUDAN UNIVERSITY · May 7, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment for children with retinoblastoma, a type of eye cancer that can be challenging to manage, especially when it comes back or doesn’t respond to previous treatments. The researchers are testing a medication called Topotecan, which is delivered directly into the eye. This study aims to see if using a higher dose of Topotecan can help shrink the tumors while also aiming to preserve the child’s vision.

To be eligible for this trial, participants must be between 1 and 12 years old and have a specific genetic mutation linked to retinoblastoma, with active tumors in one eye. They should not have severe health problems that would require immediate removal of the affected eye and must have normal kidney and liver function. If chosen to participate, families can expect regular follow-ups to monitor the child's response to the treatment and any side effects. This trial is crucial as it seeks to find a safer and more effective way to treat this serious condition while keeping the eye intact.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Patients with retinoblastoma with a somatic mutation of the gene RB1 and active tumor in a single eye, or germinal mutation of RB1 with active tumor/s in an eye and the contralateral eye unaffected, enucleated or without tumor activity. In both cases, relapsed or refractory vitreous seeding with the use of systemic, intraarterial or intravitreal chemotherapy or radiotherapy, in accordance with the availability at his/her referral site, in whom enucleation is the only recommended treatment under opinion of the medical team treating the case at the originating referral site. But the patient has a strong desire to preserve the eye.
• • 2. Normal renal function: serum creatinine: \< 45 μmol/L (0-2 years); \< 57 μmol/L (3-6 years); \< 60 μmol/L (7-10 years); \< 80 μmol/L (11-13 years).
• • 3. Normal Hepatic function: serum ALT: \< 0,52 μkat/L (de 9 months -12 years); serum AST: 61-80 g/L (8 months-5 years); 63-83 g/L (5-9 years); 63-82 g/L (9-12 years).
• • 4. Adequate marrow reserve manifested in an absolute neutrophil count \> 1000 / mm3, platelets \> 100,000 / mm3 and hemoglobin\> 8 g / dl, without transfusional or cytokine support at least one month prior to study entry.
• • 5. Age greater than 1 year and less than 12 years at the time of inclusion in the study.
• • 6. Sign the informed consent form and be willing to follow up at the specified time.
• Exclusion Criteria:
• • 1. Presence of factors that require immediate enucleation of the affected eye such as glaucoma, rubeosis iridis, anterior chamber involvement.
• • 2. Comorbidities: Uncontrolled epilepsy with anticonvulsant treatment, cardiac disease not compensated by treatment.
• • 3. Active Infections.
• • 4. Other chronic or active acute diseases that under the criterion of the researcher were an exclusion criterion.
• • 5. History of having received attenuated or live vaccines in the 30 days prior to inclusion in the study.
• • 6. Any cause of Immunosuppression.
• • 7. Trilateral Retinoblastoma.
• • 8. Extraocular spread.
• • 9. History of having received treatment for retinoblastoma with chemotherapy or radiation therapy by any means within 30 days prior to inclusion in the study.
• • 10. Patients who can not complete the study procedures for reasons psychologically or socially