Clinical trial · Interventional
Protocol for the Study and Treatment of Participants With Intraocular Retinoblastoma
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
The primary objective of this protocol is to evaluate the response rate of bilateral disease participants who have at least one eye with advanced intra-ocular retinoblastoma (stratum B) using upfront therapy with chemotherapy delivered directly to the eye. The main biology objective is to improve our understanding of the biology and tumorigenesis (how tumor develops) of retinoblastoma when biology specimens are available. As clinicians, the primary goal of the investigators for children with retinoblastoma is to provide optimal therapy using multiple treatment approaches \[chemotherapy (into the vein and directly into membrane of eyeball), cryotherapy (freeze and destroy tumor), thermotherapy (laser or heat to destroy tumor), radiation therapy, and surgical removal of eye if needed) in an attempt to preserve the eye and vision whenever possible, while still curing the disease. Therefore, all children with non-metastatic retinoblastoma at St. Jude will be offered enrollment on this study. PRIMARY OBJECTIVE: * To evaluate the response (complete + partial response) rate of bilateral disease participants who have at least one eye with advanced intraocular retinoblastoma (Stratum B) to two upfront courses of therapy consisting of subconjunctival carboplatin and systemic topotecan. SECONDARY OBJECTIVES: * To evaluate the ocular survival of eyes and event-free survival of participants by strata. * To prospectively analyze intraocular disease tissue for participants with at least one eye undergoing enucleation in order to identify the mechanism of RB1 bi-allelic inactivation. Participants may undergo upfront enucleation (due to advanced disease at diagnosis) or may receive enucleation due to progressive disease during protocol therapy.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Retinoblastoma | Retinoblastoma | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (12)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| carboplatin | Drug | Carboplatin | ALIAS |
| cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| doxorubicin | Drug | Doxorubicin | ALIAS |
| enucleation | Procedure | — | UNRESOLVED |
| etoposide | Drug | Etoposide | ALIAS |
| external beam radiation or proton beam radiation | Radiation | — | UNRESOLVED |
| filgrastim | Drug | Filgrastim | ALIAS |
| focal therapy | Other | — | UNRESOLVED |
Design
Arms and outcomes
Arms (4)
- type
- EXPERIMENTAL
- label
- Stratum A
- description
- Participants with early bilateral or unilateral (unifocal or multifocal) retinoblastoma (R-E I-III, IC A-B; R-E IV with IC A or B; or IC C with limited sub-retinal seeding), and participants with bilateral disease in whom the advanced eye has been enucleated upfront (without any high risk histopathology) and the remaining eye has early stage disease (as defined above). Interventions (see detailed description): vincristine, carboplatin, topotecan, filgrastim or PEG-filgrastim, and focal therapy, including cryotherapy, laser photocoagulation, thermo-therapy, plaque radiotherapy.
- interventionNames
- Drug: vincristine
- Drug: topotecan
- Drug: filgrastim
- Drug: PEG-filgrastim
- Drug: carboplatin
- Other: focal therapy
- type
- EXPERIMENTAL
- label
- Stratum B
- description
- Participants considered candidates for conservative management including those: 1. Participants with bilateral retinoblastoma who have R-E IV-V and IC D in one eye 2. Participants with advanced unilateral (unifocal or multifocal) retinoblastoma (R-E IV-V and IC D-E) who demonstrate foveal sparing by the tumor during EUA. Due to foveal sparing, these patients have potential for vision preservation. Interventions (see Detailed Description): vincristine, topotecan, carboplatin, etoposide, filgrastim or PEG-filgrastim and focal therapy, including cryotherapy, laser photocoagulation, thermo-therapy, plaque radiotherapy.
Eligibility
Eligibility (as posted)
- Sex
- All
Show eligibility criteria text
Inclusion Criteria: * Newly diagnosed, untreated intraocular retinoblastoma. Participants previously diagnosed with unilateral retinoblastoma treated surgically, with focal therapy or needing chemotherapy who develop asynchronous involvement of the contralateral eye, or patients with unilateral retinoblastoma treated only with enucleation or focal therapy who develop asynchronous involvement of the contralateral eye, will be eligible for study. * ECOG Performance Score must be ≤ 2 within two weeks prior to registration. * Participants must have an adequate liver function, as defined by bilirubin ≤ to 3X upper limit of normal (ULN), and SGOT and SGPT ≤ to 3X ULN. * Participants must have adequate renal function as defined by serum creatinine ≤ to 3X ULN for age. * Legal guardians must sign an informed consent indicating that they are aware of this study, the possible benefits, and toxic side effects. Legal guardians will be given a signed copy of the consent form. Exclusion Criteria: * Previously treated participants. * Presence of metastatic disease or gross (residual) orbital involvement * Participants must not have an invasive infection at time of protocol entry. * Inability or unwillingness of research participant or legal guardian/representative to give written informed consent.
References
Publications (0)
Data not yet available