Clinical trial Recruiting at 1 site Phase 3 Interventional

Using topotecan and melphalan to treat retinoblastoma

Official title: Choice of Topotecan or Melphalan in Retinoblastoma Patients

Run by Sun Yat-sen University · NCT04799002 · Data updated 2026-11-12

This study is testing if giving the chemotherapy drugs topotecan and melphalan directly into the eye can effectively treat retinoblastoma while saving the eye from removal.

Quick facts

PhasePhase 3
Study typeInterventional
SexAll
Enrollment50 (estimated)
SponsorSun Yat-sen University
Academic / other
Locations1 site (Guangzhou, Guangdong)
StatusRecruiting
StartedMarch 11, 2021
Main results expectedDecember 30, 2024
Study endsDecember 30, 2027
Registry record updatedMarch 16, 2021
Trial IDNCT04799002 on ClinicalTrials.gov

Is this trial a fit for you?

Likely a good fit

Ideal candidates for this study are patients diagnosed with retinoblastoma who are receiving eyeball-sparing treatment.

Probably not a fit

Patients whose disease has progressed during follow-up may not benefit from this study.

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study evaluates the effectiveness and potential complications of administering topotecan and melphalan through intraocular injection in patients diagnosed with retinoblastoma. It aims to provide a treatment option that spares the eyeball while addressing the cancer. The study is designed as a Phase 3 interventional trial, focusing on the outcomes of these chemotherapy agents in a specific patient population.

Why it matters

Potential benefit: If successful, this treatment could provide a less invasive option for retinoblastoma patients, preserving their eyesight while effectively managing the disease.

How similar studies have gone: While this approach is being evaluated in this specific context, similar studies using chemotherapy agents for retinoblastoma have shown promise, indicating potential for success.

Who can join: the full rules

Show full inclusion / exclusion criteria
Inclusion Criteria:

* diagnosed as retinoblastoma
* receiving eyeball-sparing treatment

Exclusion Criteria:

* disease progression during follow-up

Where it's running

  • Sun Yat-sen University
    Guangzhou, Guangdong, China
    Recruiting

Study contacts

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

How to take part

  1. Review the eligibility criteria above with your treating physician.
  2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
  3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.
Last reviewed 2026-11-14 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.