New treatments for retinoblastoma to improve vision outcomes

Is this trial a fit for you?

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

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.

Why it matters

Who can join: the full rules

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 it's running

• La Reunion - Chr Felix Guyon
  Saint-Denis, La Réunion, France Recruiting
• Amiens Chu
  Amiens, France Recruiting
• Angers Chu
  Angers, France Recruiting
• BESANCON CHU Hopital Jean Minjoz
  Besançon, France Recruiting
• Bordeaux Chu
  Bordeaux, France Recruiting
• BREST CHRU Hopital Morvan
  Brest, France Recruiting

Study contacts

• Principal investigator: Livia LUMBROSO LE ROUIC, MD — Institut Curie
• Study coordinator: Livia LUMBROSO LE ROUIC, MD
• Email: livia.lumbroso-lerouic@curie.fr
• Phone: +33(0)144324163

Similar studies

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

• High-dose Topotecan treatment for recurrent retinoblastoma with vitreous seeds

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

  Recruiting Early Phase 1 Eye & ENT Hospital of Fudan University · Shanghai, China · NCT06972602

• Linking RB1 genetic changes to eye tumors and metastasis

  We will use targeted long-read genetic sequencing on people with retinoblastoma to see if specific RB1 mutations match tumor features and the risk of spread.

  Recruiting University of Washington · Seattle, Washington · NCT06725173

• Treatment for advanced retinoblastoma using a chemoplaque

  This study is testing a new treatment using a special chemotherapy patch for young children with advanced retinoblastoma to see if it helps fight the cancer while keeping their vision safe.

  Recruiting at 2 sites Phase 2 Targeted Therapy Technologies, LLC · New York, New York and 1 other locations · NCT06679634

• New treatment for children with hard-to-treat retinoblastoma

  This study is testing a new drug for children with hard-to-treat eye cancer to see if it works better and has fewer side effects than standard chemotherapy.

  Recruiting Early Phase 1 Eye & ENT Hospital of Fudan University · Shanghai · NCT06424301

• Understanding the incidence and outcomes of trilateral retinoblastoma

  This study looks at how common trilateral retinoblastoma is and how well patients survive, comparing data from different countries to see if there are differences based on income levels.

  Recruiting Amsterdam UMC, location VUmc · Amsterdam, North Holland · NCT06367530

• Chemotherapy for high-risk retinoblastoma after eye removal

  This study is testing if giving chemotherapy after removing the eye can help prevent high-risk retinoblastoma from coming back in children with Stage I disease.

  Recruiting Phase 3 Sun Yat-sen University · Guangzhou, Guangdong · NCT05080010

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.