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RetinoblastomaPhase 2/3RETINO 2018NCT04681417

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

A Phase 2/3 study testing Melphalan or Melphalan + Topotecan, etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy and Carboplatin administered on Day 1 in people with Retinoblastoma, run by Institut Curie at 28 sites in France.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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… Read more about this study

Who is it for?
Ages 6 months to 6 years
Conditions the study is for
Retinoblastoma
What is being tested?
Melphalan or Melphalan + Topotecan, etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy and Carboplatin administered on Day 1
Could I receive a placebo?
No, everyone receives an active treatment
How many people
225 participants
Where
28 sitesFrance
Study dates
25 Mar 2021to 20 Jan 2036
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 months to 6 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Overall study inclusion criteria:
  • Newly diagnosed retinoblastoma (RB).
  • Retinoblastoma with at least one eye eligible for conservative management.
  • Patients likely to be compliant with the study requirements and visits, including late follow-up.
  • Patients not previously treated with chemotherapy or radiotherapy for this or any other cancer.
  • Patients with no contraindication to the proposed treatments.
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  • Informed consent signed by parents or legal representative.
  • 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:
  • 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
  • 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:
  • 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
  • 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.

Who cannot join

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

Trial locations and contacts

28 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Livia LUMBROSO LE ROUIC, MD contact

+33(0)144324163

livia.lumbroso-lerouic@curie.fr

Christine FOULON, PhD contact

+33(0)147111733

drci.promotion@curie.fr

28 sites in 1 country

France28 sites, 28 recruiting

  • Amiens Chu

    Amiens, 80054

    Camille KHANFARD, MD, +33(0)322087650, camille.khanfard@chu-amiens.fr

    Camille KHANFARD, MD

    Recruiting

  • Angers Chu

    Angers, 49033

    Isabelle PELLIER, MD, +33(0)241353863, ispellier@chu-angers.fr

    Isabelle PELLIER, MD

    Recruiting

  • BESANCON CHU Hopital Jean Minjoz

    Besançon, 25030

    Sébastien KLEIN, MD, +33(0)381219212, sklein@chu-beancon.fr

    Sébastien KLEIN, MD

    Recruiting

  • Bordeaux Chu

    Bordeaux, 33076

    Céline DE BOUYN ICHER, MD, +33(0)557820434, celine.icher@chu-bordeaux.fr

    Céline DE BOUYN ICHER, MD

    Recruiting

  • BREST CHRU Hopital Morvan

    Brest, 29609

    Liana-Stéphania CARAUSU, MD, (+33) 02 98 22 37 70, liana.carausu@chu-brest.fr

    Liana-Stéphania CARAUSU, MD

    Recruiting

  • CAEN CHU

    Caen, 14033

    Damien BODET, MD, +33(0)231064488, bodet-d@chu-caen.fr

    Damien BODET, MD

    Recruiting

  • CHU ESTAING Centre Régional de Cancérologie et Therapie Cellulaire Pediatrique (CRCTCP)

    Clermont-Ferrand, 63003

    Justyna KANOLD, MD, (+33) 04 73 75 00 09, jkanold@chu-clermontferrand.fr

    Justyna KANOLD, MD

    Recruiting

  • DIJON CHU Hopital François Mitterand

    Dijon, 21079

    Claire BRIANDET, MD, +33(0)380293601, claire.briandet@chu-dijon.fr

    Claire BRIANDET, MD

    Recruiting

  • Grenoble Chu

    Grenoble, 38045

    Anne PAGNIER, MD, 04 76 76 58 93, APagnier@chu-grenoble.fr

    Anne PAGNIER, MD

    Recruiting

  • LILLE Centre Oscar Lambret

    Lille, 59037

    Hélène SUDOUR BONNANGE, MD, +33(0)320295959, h-sudour@o-lambret.fr

    Hélène SUDOUR BONNANGE, MD

    Recruiting

  • Limoges Chu

    Limoges, 87042

    Christophe PIGUET, MD, +33(0)555056801, christophe.piguet@chu-limoges.fr

    Christophe Christophe PIGUET, MD

    Recruiting

  • LYON Centre Léon Bérard

    Lyon, 69373

    BENOIT DUMONT, MD, benoit.dumont@ihope.fr

    +33 (0)4 69 16 65 74

    Recruiting

  • Marseille Chu

    Marseille, 13385

    Carole COZE, MD, +33(0)491386821, carole.coze@ap-hm.fr

    Carole COZE, MD

    Recruiting

  • MONTPELLIER CHU Hopital Arnaud De Villeneuve

    Montpellier, 34295

    Nicolas SIRVENT, MD, +33(0)467336519, n-sirvent@chu-montpellier.fr

    Nicolas SIRVENT, MD

    Recruiting

  • NANTES CHU Hopital Mere-Enfant

    Nantes, 44093

    LECULEE THEBAUD Estelle, MD, +33(0)240083610, estelle.thebaud@chu-nantes.fr

    LECULEE THEBAUD Estelle, MD

    Recruiting

  • NICE CHU Hopital Archet 2

    Nice, 06202

    Gwenaëlle DUHIL DE BENAZE, MD, +33(0)492036064, duhildebenaze.g@chu-nice.fr

    Gwenaëlle DUHIL DE BENAZE, MD

    Recruiting

  • Institut Curie

    Paris, 75005

    Livia LUMBROSO LE ROUIC, MD, +33(0)144324163, livia.lumbroso-lerouic@curie.fr

    Isabelle AERTS, MD, +33(0)144324333, isabelle.aerts@curie.fr

    Recruiting

  • PARIS Fondation Ophtalmologique Adolphe de Rothschild

    Paris, 75019

    Raphaël BLANC, MD, +33(0)148036828, rblanc@for.paris

    Raphaël BLANC, MD

    Recruiting

  • Poitiers Chu

    Poitiers, 86021

    Frédéric MILLOT, MD, +33(0)549443078, f.millot@chu-poitiers.fr

    Frédéric MILLOT, MD

    Recruiting

  • Reims Chu

    Reims, 51092

    Claire PLUCHART, MD, +33(0)326787515, cpluchart@chu-reims.fr

    Claire PLUCHART, MD

    Recruiting

  • Rennes Chu

    Rennes, 35056

    Chloé PUISEUX, MD, +33(0)299265917, chloe.puiseux@chu-rennes.fr

    Chloé PUISEUX, MD

    Recruiting

  • Rouen Chu

    Rouen, 76000

    Cécile DUMESNIL DE MARICOURT, MD, +33(0)232888191, cecile.dumesnil@chu-rouen.fr

    Cécile DUMESNIL DE MARICOURT, MD

    Recruiting

  • La Reunion - Chr Felix Guyon

    Saint-Denis, La Réunion, 97405

    Yves REGUERRE, MD, 26(2)262905676, yves.reguerre@chu-reunion.fr

    Yves REGUERRE, MD

    Recruiting

  • Saint Etienne Chu

    Saint-Etienne, 42055

    Jean-Louis STEPHAN, MD, +33(0)478828608, j.louis.stephan@chu-st-etienne.fr

    Jean-Louis STEPHAN, MD

    Recruiting

  • Strasbourg Chu

    Strasbourg, 67098

    Natacha ENTZ WERLE, MD, +33(0)388128396, natacha.entz-werle@chu-strasbourg.fr

    Natacha ENTZ WERLE, MD

    Recruiting

  • Toulouse Chu

    Toulouse, 31026

    Anne-Isabelle BERTOZZI-SALAMON, MD, +33(0)534558613, bertozzi.ai@chu-toulouse.fr

    Anne-Isabelle BERTOZZI-SALAMON, MD

    Recruiting

  • TOURS CHU Hopital Clocheville

    Tours, 37044

    Pascale BLOUIN, MD, +33(0)247474751, p.blouin@chu-tours.fr

    Pascale BLOUIN, MD

    Recruiting

  • Nancy Chu

    Vandœuvre-lès-Nancy, 54500

    Ludovic MANSUY, MD, +33(0)383154734, lu.mansuy@chu-nancy.fr

    Ludovic MANSUY, MD

    Recruiting

Other trials in France

About this study

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

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.
Read the detailed description

Primary objective of the studies:

  • Study 1: To evaluate the efficacy of IAC in term of local control of the disease in patients eligible for IAC (randomized phase II study);
  • Study 2: To assess the visual function, based on World Health Organization (WHO) criteria, in patients eligible for other conservative treatments (IV chemotherapy in association with local ophthalmologic trématent or local ophthalmological treatment without IV chemotherapy; this is a minimally invasive interventional study).
Conditions
Retinoblastoma
Being tested
Melphalan or Melphalan + Topotecan, etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy, Carboplatin administered on Day 1, Thermotherapy (local treatment), Cryotherapy (local treatment)
Keywords
Pediatric, Ocular conservative treatment in retinoblastoma
Medical subject headings
Retinoblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Melphalan or Melphalan + TopotecanDrug

    Also called Alkeran, Topotecan Accord; Topotecan Hospira chemotherapy

    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 chemotherapyDrug

    Also called etoposide, carboplatin and vincristine 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 1Drug

    Also called platinum-based chemotherapy

    Chemothermotherapy : Intravenous injection by carboplatin

  • Thermotherapy (local treatment)Device

    Also called Thermotherapy after carboplatin administered on Day 1

    Thermotherapy after carboplatin administered on Day 1

  • Cryotherapy (local treatment)Device

    Cryotherapy (local treatment)

  • Iodine-125 plaques (local treatment)Device

    Iodine-125 plaques (local treatment)

  • Intravitreal Melphalan chemotherapy injections (local treatment)Drug

    Intravitreal Melphalan chemotherapy injections (local treatment)

Study groups in NCT04681417
Study groupRoleWhat they receive
Study 1: Melphalan or Melphalan + TopotecanExperimentalRandomized phase-II study evaluating the efficacy of Intra-Arterial Chemotherapy (IAC) with melphalan and topotecan versus melphalan alone, both in association with ophthalmologic treatments.
Receives: Drug: Melphalan or Melphalan + Topotecan; Device: Thermotherapy (local treatment); Device: Cryotherapy (local treatment); Device: Iodine-125 plaques (local treatment); Drug: Intravitreal Melphalan chemotherapy injections (local treatment)
Study 2: Etoposide, carboplatin and vincristineOtherNeoadjuvant chemotherapy involves 2 to 6 cycles of combined etoposide, carboplatin and vincristine in association with ophthalmologic treatments.
Receives: Drug: etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy; Drug: Carboplatin administered on Day 1; Device: Thermotherapy (local treatment); Device: Cryotherapy (local treatment); Device: Iodine-125 plaques (local treatment); Drug: Intravitreal Melphalan chemotherapy injections (local treatment)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Study 1: Rate of eye preservation

    Rate of eye preservation in the 2 treatments' arms

    Measured: 24 months after randomization

  2. Visual function

    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

    Measured: when patient is 6 years old and at least 24 months of follow-up after study inclusion

Secondary outcomes (7)

  1. Ocular toxicity

    Percentage of patients with at least one grade-3 or -4 toxicity (CTCAE v. 5.0)

    Measured: Up to 24 months after inclusion in the study

  2. Systemic toxicity

    Rate of specific grade 3-4 toxicity (CTCAE v. 5.0)

    Measured: Up to 24 months after inclusion in the study

  3. Relapse

    Percentage of patients with relapse within 24 months after inclusion, broken down into: Local relapse, Extra-ocular relapse

    Measured: within 24 months after inclusion

  4. Second malignant tumor

    Percentage of patients with second malignant tumor within 24 months after study inclusion

    Measured: 24 months after study inclusion

  5. Additional assessment of visual function

    Additional assessment of visual function at follow-up: Percentage of patients at each of the four levels of visual acuity according to WHO criteria

    Measured: In the year patients turn 6 years of age and at least 24 months of treatment

  6. Retinal assessment

    Optical Coherence Tomography (OCT) and Optical Coherence Tomography-Angiography (OCT-A) measures on retina

    Measured: In the year patients turn 6 years of age and at least 24 months of treatment

  7. Integration at school

    Rate of children able to attend the common primary school : without specific help or with the help of a dedicated person and/or with specific features to facilitate reading and writing

    Measured: In the year patients turn 6 years of age and at least 24 months of treatment

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Dec 2020
  2. Study start25 Mar 2021
  3. Primary completion20 Jan 2035 (estimated)
  4. Study completion20 Jan 2036 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
225 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04681417
Sponsor's protocol ID
IC 2019-05
Acronym
RETINO 2018
Status last verified
Nov 2025
First submitted
17 Dec 2020
Last updated
28 Nov 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Sponsor will share de-identified data sets Documents generated under the project will be disseminated in accordance with Institut Curie policies.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Melphalan or Melphalan + Topotecan, etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy and Carboplatin administered on Day 1 in people with Retinoblastoma, run by Institut Curie at 28 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 16 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 28 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 28 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Livia LUMBROSO LE ROUIC, MD (+33(0)144324163, livia.lumbroso-lerouic@curie.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Study 1: Rate of eye preservation (time frame: 24 months after randomization). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04681417.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Nov 2025.

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