Using topotecan and melphalan to treat retinoblastoma

Choice of Topotecan or Melphalan in Retinoblastoma Patients

Phase 3 Interventional Sun Yat-sen University · NCT04799002

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
Enrollment50 (estimated)
SexAll
SponsorSun Yat-sen University Academic / other
Locations1 site (Guangzhou, Guangdong)
Trial IDNCT04799002 on ClinicalTrials.gov

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.

Who should consider this trial

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

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

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

Eligibility criteria

Show full inclusion / exclusion criteria
Inclusion Criteria:

* diagnosed as retinoblastoma
* receiving eyeball-sparing treatment

Exclusion Criteria:

* disease progression during follow-up

Where this trial is running

Guangzhou, Guangdong

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions RetinoblastomaChemotherapy EffecttopotecanMelphalan
Last reviewed 2026-06-13 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.