New chemo cocktails aim to save eyes in kids with retinoblastoma
NCT ID NCT02866136
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This phase 2 trial tests different chemotherapy regimens in 133 children with retinoblastoma, a rare eye cancer. The goal is to shrink tumors and avoid removing the eye or using radiation. Treatments include intravenous and intra-arterial chemotherapy, sometimes combined with laser therapy.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- chemotherapy (etoposide, carboplatin, melphalan, vincristine)
- What this could lead to
- If successful, this could offer effective eye-sparing treatments for children with retinoblastoma, reducing the need for eye removal or radiation.
- What could go wrong
- This is a mid-stage trial with no control group, so results are preliminary. Chemotherapy has significant side effects, and not all children may respond well.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 133 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Feb 2012
- Expected to finish
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Sep 2035
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Up to 6 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: Study 1 inclusion criteria: * Patients affected by unilateral retinoblastoma groups A, B (according to the age), group C (according to the age and vitreous seeding),or bilateral retinoblastoma groups A, B, C (excluding the eyes with macular threat and bilateral group D eyes or on the only remaining eye) amenable to a conservative treatment (at least on one eye in bilateral disease) but needing initial chemotherapy because of the location, the size of the lesion (more than 4 mm of diameter), a vision threat or risks of intravitreal relapse making those patients not amenable to chemothermotherapy first line. * Patients less than six months of age with unilateral retinoblastoma groups B, C, D, or bilateral very asymmetric with one eye group D and the other amenable to local treatments without chemotherapy. * Children from 0 to 6 years old. Study 2 inclusion criteria: * Patients affected by unilateral or bilateral retinoblastoma group B (according to the age), group C (according to the age and vitreous seeding), or group D. * Patients affected by bilateral retinoblastoma very asymmetric with a group D eye that can be treated by intraarterial chemotherapy by Melphalan and the other amenable to local treatments without chemotherapy. * Children from 6 months to 6 years old. Study 3 inclusion criteria: * Children affected of bilateral group D retinoblastoma or on the only eye amenable to conservative treatment. * Children from 0 to 6 years old. Common inclusion criteria: * Patients not previously treated by chemotherapy or radiotherapy for this tumour or another cancer. * No contra-indications to the study treatments * Possible long term follow-up. * Written informed consent of the parents or the legal representative. * Patients having social security cover. Exclusion Criteria: Study 1 exclusion criteria: * Patients for whom a local treatment is possible without initial chemotherapy (tumour smaller than 4 mm and located far from optic nerve head or macula). * Patients with an unilateral group D with massive tumour or group E eyes needing enucleation first line or after initial chemotherapy (in case of buphthalmia or suspected optic nerve invasion or extrascleral extension). * Patients affected by bilateral retinoblastoma very asymmetric with a group D eye that can be treated by intraarterial chemotherapy by Melphalan and the other amenable to local treatments without chemotherapy. * Patients with bilateral retinoblastoma and bilateral group D eyes or on the only remaining eye or presenting a bilateral macular threat requiring conservative treatment by a 6 cycles, three drugs regimen. Study 2 exclusion criteria: * Patients with a unilateral group D (extensive) or B or C but covering the optic nerve head or group E eyes for which enucleation is warranted first line or after chemotherapy (in case of buphthalmia or suspected optic nerve invasion or extrascleral extension). * Patients with unilateral group D eye with tumour volume of more than 50% of eye volume, for whom a massive choroidal invasion could be associated (on clinical or imaging criteria) and for which enucleation is warranted. Study 3 exclusion criteria: * Patients for whom a local treatment is possible without chemoreduction (tumour smaller than 4 mm, distant from macula and from optic nerve head). * Patients affected by bilateral retinoblastoma very asymmetric with a group D eye that can be treated by intraarterial chemotherapy by Melphalan and the other amenable to local treatments without chemotherapy. * Patients with bilateral retinoblastoma without macular threat or groups A, B, C than can be treated with chemoreduction by VP16 and Carboplatin then chemothermotherapy without laser at day 8. Common exclusion criteria: * Patients older than 6 years old. * Patients with extraocular retinoblastoma. * Patients with a disease being a contra-indication to chemotherapy. * Patients anteriorly treated by chemotherapy. * Patients anteriorly treated by external beam irradiation. * Patients anteriorly treated for another cancer. * Follow-up not possible due to geographic distance from the center or for social or psychological reasons. * Parents not having accepted the therapeutic strategy after explanations by the investigator. * Contra-indication to the use of one of the drugs used in the study.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Institut Curie
Paris, 75005, France
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Other studies related to the condition(s) this trial covers.
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