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New treatments for retinoblastoma to improve vision outcomes

Ocular Conservative Treatment for Retinoblastoma: Efficacy of the New Management Strategies and Visual Outcome - RETINO 2018

PHASE2; PHASE3 · Institut Curie · NCT04681417

This study is testing new treatment options for children with retinoblastoma to see if they can improve vision and control the disease better than current methods.

Quick facts

PhasePHASE2; PHASE3
Study typeInterventional
Enrollment225 (estimated)
Ages6 Months to 6 Years
SexAll
SponsorInstitut Curie (other)
Drugs / interventionschemotherapy
Locations28 sites (Saint-Denis, La Réunion and 27 other locations)
Trial IDNCT04681417 on ClinicalTrials.gov

What this trial studies

This clinical trial consists of two independent multicenter studies aimed at evaluating different therapeutic approaches for retinoblastoma in pediatric patients. The first study is a randomized phase II trial comparing the efficacy of intra-arterial chemotherapy with melphalan and topotecan against melphalan alone, both combined with ophthalmologic treatments. The second study focuses on assessing visual function in patients receiving intravenous chemotherapy alongside local ophthalmological treatments or local treatments without chemotherapy. Both studies aim to enhance local disease control and improve visual outcomes in children diagnosed with retinoblastoma.

Who should consider this trial

Good fit: Ideal candidates include children aged 6 months to 6 years with newly diagnosed retinoblastoma eligible for conservative management.

Not a fit: Patients who have previously received chemotherapy or radiotherapy for retinoblastoma or any other cancer may not benefit from this study.

Why it matters

Potential benefit: If successful, this trial could lead to improved visual outcomes and better management strategies for children with retinoblastoma.

How similar studies have performed: Previous studies have shown promise in using intra-arterial chemotherapy for retinoblastoma, indicating potential success for this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

Overall study inclusion criteria:

1. Newly diagnosed retinoblastoma (RB).
2. Retinoblastoma with at least one eye eligible for conservative management.
3. Patients likely to be compliant with the study requirements and visits, including late follow-up.
4. Patients not previously treated with chemotherapy or radiotherapy for this or any other cancer.
5. Patients with no contraindication to the proposed treatments.
6. Informed consent signed by parents or legal representative.
7. French Social Security System coverage.

Study 1 inclusion criteria:

8.1. Children aged from 6 months to 6 years. 9. Retinoblastoma in at least one eye, deemed manageable with IAC in one side and without IV chemotherapy:

1. Unilateral Retinoblastoma classified as group B, C (if vitreous seeds \< 3 mm), D or E with no invasion of the anterior segment, and without massive tumor of more than 2/3 of the eye, eligible for conservative management, or
2. Bilateral Retinoblastoma but with very unbalanced lesions between the two eyes: one eye group D or E without invasion of the anterior segment or massive tumor of more than 2/3 of the eye, eligible for IAC, and the other eye eligible for local treatment only (without IAC).

Study 2 inclusion criteria:

8.2. Retinoblastoma eligible for conservative management although not manageable with IAC:

1. Unilateral Retinoblastoma in children below 6 months of age, classified as Group A, B, C or D, with or without vitreous seeding, compatible with conservative management, or
2. Bilateral Retinoblastoma classified as group A, B, C D, or E without invasion of the anterior segment, and/or massive tumors of more than 2/3 of the eye and eligible for conservative management.

Exclusion Criteria:

Overall study non-inclusion criteria:

1. RB not eligible for conservative management :

   1. Extra-ocular extension of the disease, or
   2. Group E eyes with invasion of the anterior segment, and/or massive tumors of more than 2/3 of the eye.
2. Patient older than 6 years of age.
3. Patients with another associated disease contra indicating systemic chemotherapy.
4. Previously treated retinoblastoma by chemotherapy.
5. Patients already treated for another malignant disease.
6. Patient with any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule.
7. Patients whose parents have not accepted the treatment regimen after explanation of it.
8. Contraindication to study drug mentioned in SmPC (Summary of Products Characteristics) of the study drugs.
9. Inclusion in another experimental anti-cancer drug therapy.

   Study 1 non-inclusion criteria:
10. Any contraindication or concomitant disease that would preclude the Study 1 treatment procedure and could delay treatment.

These patients should be eligible for Study 2.

Where this trial is running

Saint-Denis, La Réunion and 27 other locations

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.

View on ClinicalTrials.gov →

Conditions: Retinoblastoma, Pediatric, Ocular conservative treatment in retinoblastoma

Last reviewed 2026-05-15 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.