High-dose Topotecan treatment for recurrent retinoblastoma with vitreous seeds

High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed(A Prospective Non-controlled Single Arm Trial)

This study is testing whether high-dose Topotecan can help patients with recurrent retinoblastoma and vitreous seeds keep their eye while fighting the tumor.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 1 Year to 12 Years
• Sex: All
• Sponsor: Eye & ENT Hospital of Fudan University (Academic / other)
• Drugs / interventions: chemotherapy, radiation
• Locations: 1 site (Shanghai, China)
• Trial ID: NCT06972602 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the use of high-dose Topotecan, a chemotherapeutic agent, to treat patients with retinoblastoma who have recurrent or refractory vitreous seeds. The study aims to improve anti-tumor activity while minimizing side effects associated with traditional chemotherapy. Patients eligible for this trial include those with specific genetic mutations and active tumors in one eye, who wish to preserve their eye despite the recommendation for enucleation. The trial will also involve the collection of aqueous humor and the use of electroretinograms to assess retinal function.

Who should consider this trial

Good fit: Ideal candidates are children with retinoblastoma exhibiting specific RB1 mutations and active tumors in one eye, who have previously undergone various treatments but wish to avoid enucleation.

Not a fit: Patients with bilateral retinoblastoma or those whose tumors are not responsive to any form of chemotherapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could enhance eye preservation and improve outcomes for children with difficult-to-treat retinoblastoma.

How similar studies have performed: While there have been studies on intravitreal chemotherapy for retinoblastoma, the specific use of high-dose Topotecan in this context is novel and has not been extensively tested.

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

Where this trial is running

Shanghai, China

• Fudan Eye & ENT Hospital — Shanghai, China, China (Recruiting)

Study contacts

• Study coordinator: Kang Xue, MD

Conditions Retinoblastoma; Retinoblastoma, Recurrent