Ireland
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Recruiting Phase 2/3

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

NCT04681417 · tracked via the Priya Life Science France tracker
Phase
Phase 2/3
Started
2021-03-25
Last updated
2025-11-28

Condition(s) studied

Retinoblastoma

Investigational drug(s) / intervention(s)

Melphalan or Melphalan + TopotecanAll Melphalan trials (32) →All Topotecan trials (52) →etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy →Carboplatin administered on Day 1Thermotherapy (local treatment)Cryotherapy (local treatment)Iodine-125 plaques (local treatment)Intravitreal Melphalan chemotherapy injections (local treatment)

Melphalan or Melphalan + Topotecan: IAC: 2-6 cycles every 1 month (number based on the local tumor evolution), performed using radio-guided catheterization and delivery at the ostium of the ophthalmic artery, in neuroradiology operating room, and under General Anesthesia (GA). IAC will be administered at the Fondation Rothschild Hospital in collaboration with the Interventional Neuroradiology team.

etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy: 2 to 6 cycles of combined etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy : thermotherapy and/or cryotherapy and/or iodine-125 plaques and/or intravitreal chemotherapy injections

Carboplatin administered on Day 1: Chemothermotherapy : Intravenous injection by carboplatin

Thermotherapy (local treatment): Thermotherapy after carboplatin administered on Day 1

Cryotherapy (local treatment): Cryotherapy (local treatment)

Iodine-125 plaques (local treatment): Iodine-125 plaques (local treatment)

Intravitreal Melphalan chemotherapy injections (local treatment): Intravitreal Melphalan chemotherapy injections (local treatment)

Study summary

This protocol includes 2 independent studies. Both studies are multicenter studies, evaluating different therapeutic approaches in two different populations of patients:

* Study 1, a randomized phase-II study evaluating the efficacy of Intra-Arterial Chemotherapy (IAC) with melphalan and topotecan versus melphalan alone, both in association with ophthalmologic treatments.
* Study 2, a minimally invasive interventional study evaluating the results on visual function following reference treatment with intravenous (IV) chemotherapy in association with ophthalmologic or local ophthalmological treatment without IV chemotherapy.

Eligibility

Sex
ALL
Min age
6 Months
Max age
6 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

  • Study 1: Rate of eye preservation — 24 months after randomization
    Rate of eye preservation in the 2 treatments' arms
  • Visual function — when patient is 6 years old and at least 24 months of follow-up after study inclusion
    Percentage of patients with major, mild or no impairment of visual function according to WHO criteria, i.e. normal bilateral visual acuity (≥ 6/10) or mild bilateral visual impairment (3/10 to 5/10) according to tumor location and extension

Trial sites (28)

FacilityCityRegionStatus
La Reunion - Chr Felix Guyon Saint-Denis La Réunion 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
CAEN CHU Caen France Recruiting
CHU ESTAING Centre Régional de Cancérologie et Therapie Cellulaire Pediatrique (CRCTCP) Clermont-Ferrand France Recruiting
DIJON CHU Hopital François Mitterand Dijon France Recruiting
Grenoble Chu Grenoble France Recruiting
LILLE Centre Oscar Lambret Lille France Recruiting
Limoges Chu Limoges France Recruiting
LYON Centre Léon Bérard Lyon France Recruiting
Marseille Chu Marseille France Recruiting
MONTPELLIER CHU Hopital Arnaud De Villeneuve Montpellier France Recruiting
NANTES CHU Hopital Mere-Enfant Nantes France Recruiting
NICE CHU Hopital Archet 2 Nice France Recruiting
Institut Curie Paris France Recruiting
PARIS Fondation Ophtalmologique Adolphe de Rothschild Paris France Recruiting
Poitiers Chu Poitiers France Recruiting
Reims Chu Reims France Recruiting
Rennes Chu Rennes France Recruiting
Rouen Chu Rouen France Recruiting
Saint Etienne Chu Saint-Etienne France Recruiting
Strasbourg Chu Strasbourg France Recruiting
Toulouse Chu Toulouse France Recruiting
TOURS CHU Hopital Clocheville Tours France Recruiting
Nancy Chu Vandœuvre-lès-Nancy France Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04681417 on ClinicalTrials.gov ↗ ← All trials in France