RetinoblastomaPhase 2NCT01783535
Protocol for the Study and Treatment of Participants With Intraocular Retinoblastoma
A Phase 2 study testing vincristine, topotecan and filgrastim in people with Retinoblastoma, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Retinoblastoma
- What is being tested?
- vincristine, topotecan and filgrastim
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 174 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Jun 2013to 21 Feb 2027
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
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:
Read more
- 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.
Read the detailed description
Participants will be stratified into four main treatment groups, depending on whether retinoblastoma is present in one or both eyes and disease grouping [early or advanced, Reese-Ellsworth (R-E) group I-V, and International Classification A-E]. Additionally, participants will be invited to participate in exploratory research objectives that address cognitive and functional development of children with retinoblastoma, the pharmacokinetics of topotecan in young children, and evaluation of ototoxicity, including genetic analysis.
TREATMENT PLAN
STRATUM A:
- Children ≥ or equal to 6 months old at time of enrollment - 8 courses of vincristine and carboplatin, given at 3-4 week intervals.
- Infants < 6 months old at time of enrollment - Therapy will consist of six courses of chemotherapy; three courses of vincristine and carboplatin, given at 3-4 week intervals, alternating with 3 cycles of vincristine and topotecan, given at 3-4 week intervals.
Focal treatments will be administered at the discretion of the treating team. Focal therapies will include cryotherapy, laser photocoagulation, thermo-therapy and plaque radiotherapy.
STRATUM B:
- Participants without extensive sub-retinal (SR) seeding, treatment will consist of two up-front courses of vincristine and topotecan, given at 3-4 week intervals.
- Participants without SR seeding: ≥ or equal to partial response after 2 cycles, will receive three additional courses of vincristine-topotecan (VT) and six courses of vincristine-carboplatin, given at 3-4 week intervals.
- Participants without SR seeding and < partial response after 2 cycles VT will receive 6 courses of vincristine-carboplatin-etoposide (VCE), given at 3-4 week intervals.
- Participants with extensive sub-retinal (SR) seeding will receive two up-front courses of subconjunctival (also called subtenon or periocular) CARBOplatin and systemic topotecan, given at 3-4 week intervals.
- Participants with SR seeding: > or equal to partial response after 2 cycles will receive three additional courses of vincristine-topotecan, and six courses of vincristine-carboplatin, given at 3-4 week intervals.
- Participants with SR seeding: < partial response after 2 cycles will receive 6 courses of VCE, given at 3-4 week intervals.
Focal treatments will be administered at the discretion of the treating team. Focal therapies will include cryotherapy, laser photocoagulation, thermo-therapy and plaque radiotherapy.
STRATUM C:
Participants with unilateral (unifocal or multifocal) advanced (R-E IV-V and IC D-E) intraocular disease will undergo enucleation. Adjuvant therapy will be based on histopathology:
- low risk participants: Participants in whom the enucleated eye does not show extra-retinal disease (see definition of intermediate and high risk below); will not receive any additional treatment.
- intermediate risk participants: Participants in whom the enucleated eye shows presence of tumor in the anterior chamber, invasion of the ciliary body/iris, massive invasion of the choroid, and invasion of the optic nerve beyond the lamina cribrosa with concomitant invasion of the choroid, will receive 4 courses of adjuvant chemotherapy with vincristine-carboplatin-doxorubicin (VCD).
- high risk participants: Participants in whom the enucleated eye shows involvement of the sclera, or involvement of the optic nerve at the level of the cut-end, will be treated with 6 courses of chemotherapy, with alternating courses of VCE and VCD.
- High-risk participants with extra-ocular extension (i.e. tumor extending beyond the sclera/cornea or beyond the cut end of the optic nerve) will be candidates for external-beam radiation therapy (EBRT) to the entire orbit, including the optic nerve, administered after 2 or 3 courses of treatment. Patients with extra-ocular extension may be considered for enrollment on an alternative therapeutic protocol for metastatic retinoblastoma (or best clinical management).
STRATUM D:
Management of participants with bilateral retinoblastoma is often complex; and some participants will have one eye enucleated upfront due to advanced disease. The decision for enucleation will be made after thorough consideration by the treating team. The treatment of the remaining eye will depend on a combination of two factors: a) R-E group of the remaining eye, and b) Histology of the enucleated eye. Though we have accumulated some information regarding the use of vincristine, cyclophosphamide, and doxorubicin in the treatment of intraocular retinoblastoma, it is not considered standard of care. Therefore, participants with intermediate and high risk features will be treated with 6 courses of vincristine, carboplatin, and etoposide (VCE). Those participants in whom the enucleated eye shows only low risk histology will be eligible to proceed with either stratum A or stratum B therapy. For those receiving stratum B therapy, consideration of periocular carboplatin will be allowed. External beam or proton beam radiation therapy will be considered for patients with extra-ocular extension.
Focal treatments will be administered at the discretion of the treating team. Focal therapies will include cryotherapy, laser photocoagulation, thermo-therapy and plaque radiotherapy.
- Conditions
- Retinoblastoma
- Being tested
- vincristine, topotecan, filgrastim, PEG-filgrastim, carboplatin
- Keywords
- intraocular retinoblastoma
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vincristineDrug
Also called VCR, Oncovin(R)
Given via minibag/gravity flow.
topotecanDrug
Also called TOPO, Hycamtin(R)
Given IV over 30 minutes.
filgrastimDrug
Also called G-CSF, Neupogen(R)
Given subcutaneously 24-36 hours after chemotherapy for 7-10 days, until absolute neutrophil count (ANC) is >2,000/µL on one occasion after the expected nadir.
PEG-filgrastimDrug
Also called pegylated filgrastim, PEG filgrastim, SD-01, Neulasta(R)
Given subcutaneously 24-36 hours after chemotherapy for 7-10 days, until ANC is >2,000/µL on one occasion after the expected nadir.
carboplatinDrug
Also called CARBO, Paraplatin(R)
Given IV over 60 minutes. Given periocular (subtenon/subconjunctival).
focal therapyOther
Also called cryotherapy, laser photocoagulation, thermo-therapy, plaque radiotherapy
Focal treatments will be administered at the discretion of the treating team to strata A, B and D. In select cases of very early stage retinoblastoma (Stratum A), participants may receive focal therapies only and chemotherapy will be held at the discretion of the treating team. If there is any evidence of progression or unsatisfactory results, the participant will begin chemotherapy as per Stratum A. For selected participants an effort will be made to perform sequential chemo-thermotherapy. In these cases, carboplatin will be administered one or two hours prior to thermotherapy.
etoposideDrug
Also called ETOP, VP-16, Vepesid(R), Etopophos(R)
Given IV. Participants who cannot tolerate etoposide may be given etoposide phosphate (Etopophos(R)).
cyclophosphamideDrug
Also called CYCLO, Cytoxan(R)
Given IV.
MESNADrug
Also called Mesnex(R)
Given IV before CYCLO and at 3, 6 and 9 hours after CYCLO.
doxorubicinDrug
Also called DOXO, Adriamycin(R)
Given IV on Day 1 of Cycles 2, 4 and 6 in Stratum C high-risk.
enucleationProcedure or surgery
Also called eye removal
Eye removal due to advanced disease in Strata C and D participants.
external beam radiation or proton beam radiationRadiation
Also called EBRT
EBRT or proton beam radiation will be administered to any eye in which the disease is considered to be not controllable with focal treatments alone, and in participants with enucleated eyes in which high risk of orbital and/or central nervous system disease is documented histologically (high-risk group with disease extension beyond the sclera or cornea, or beyond the cut end of the optic nerve). EBRT will be administered using standard techniques practices with the objective of limiting dose to normal tissues including the hypothalamic-pituitary unit, supratentorial brain, orbit, cochleae and contralateral eye when indicated. Participants will be evaluated on an individual basis to determine whether they might benefit from referral for proton therapy.
| Study group | Role | What they receive |
|---|---|---|
| Stratum A | Experimental | 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. Receives: Drug: vincristine; Drug: topotecan; Drug: filgrastim; Drug: PEG-filgrastim; Drug: carboplatin; Other: focal therapy |
| Stratum B | Experimental | 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. Receives: Drug: vincristine; Drug: topotecan; Drug: filgrastim; Drug: PEG-filgrastim; Drug: carboplatin; Other: focal therapy; Drug: etoposide |
| Stratum C | Experimental | Participants with advanced (R-E IV-V and IC D-E) unilateral retinoblastoma who require upfront enucleation. Participants will be assessed and treated by low, intermediate or high risk.
Interventions (see Detailed Description): vincristine, cyclophosphamide, MESNA, doxorubicin, etoposide, carboplatin, filgrastim or PEG-filgrastim, enucleation Receives: Drug: vincristine; Drug: filgrastim; Drug: PEG-filgrastim; Drug: carboplatin; Drug: etoposide; Drug: cyclophosphamide; Drug: MESNA; Drug: doxorubicin; Procedure: enucleation |
| Stratum D | Experimental | Participants with bilateral retinoblastoma who may require upfront enucleation for one eye due to advanced disease (R-E IV-V and IC E).
Interventions (see Detailed Description): vincristine, carboplatin, topotecan, etoposide, enucleation, filgrastim or PEG-filgrastim, focal therapy, including cryotherapy, laser photocoagulation, thermotherapy (and thermo-chemotherapy) and episcleral plaque brachytherapy, and external beam radiation or proton beam radiation in select cases. Receives: Drug: vincristine; Drug: topotecan; Drug: filgrastim; Drug: PEG-filgrastim; Drug: carboplatin; Other: focal therapy; Drug: etoposide; Procedure: enucleation; Radiation: external beam radiation or proton beam radiation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response Rate
To evaluate the response (CR + PR) rate of bilateral disease patients who have at least one eye with advanced intraocular retinoblastoma (stratum B patients) and subretinal seeding to two upfront courses of therapy consisting of subconjunctival carboplatin and systemic topotecan. Stratum B patients who had no significant subretinal seeding and received vincristine and topotecan are not evaluable for this primary objective. CR: Complete regression, calcification or scarring of all apparent tumor masses in the funduscopic examination. Lesions which initially decrease in size and then remain unchanged (but incompletely calcified or scarred) in the absence of further focal or systemic therapy will also be considered CR. The response must persist for at least 4 weeks. PR: Greater than 50% (but less than 100%) reduction of the tumor masses in the funduscopic examination, without the appearance of any new lesions. The response must persist for at least 4 weeks.
Measured: Through completion of two upfront courses of therapy (each course lasting 3-4 weeks; total approximately 6-8 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2013 (estimated)
- Study start19 Jun 2013
- Primary completion17 Mar 2023
- Study completion21 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for RetinoblastomaStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 174 participants (actual)
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Carlos Rodriguez-Galindo, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01783535
- Sponsor's protocol ID
- SJRET6
- Other IDs
- NCI-2013-00409
- Status last verified
- Jun 2026
- First submitted
- 31 Jan 2013
- Last updated
- 29 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Protocol for the Study and Treatment of Participants With Intraocular Retinoblastoma
- ClinicalTrials.gov ID
- NCT01783535
- Acronym / sponsor ID
- SJRET6
- Conditions
- Retinoblastoma
- Keywords
- intraocular retinoblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 174 participants (actual)
- Lead sponsor
- St. Jude Children's Research HospitalUniversity, hospital or other
- Study start
- 19 Jun 2013 (actual)
- Primary completion
- 17 Mar 2023 (actual)
- Study completion
- 21 Feb 2027 (estimated)
- First posted
- 5 Feb 2013 (estimated)
- Results
- Posted 29 Jun 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Participants will be stratified into four main treatment groups, depending on whether retinoblastoma is present in one or both eyes and disease grouping [early or advanced, Reese-Ellsworth (R-E) group I-V, and International Classification A-E]. Additionally, participants will be invited to participate in exploratory research objectives that address cognitive and functional development of children with retinoblastoma, the pharmacokinetics of topotecan in young children, and evaluation of ototoxicity, including genetic analysis.
TREATMENT PLAN
STRATUM A:
- Children ≥ or equal to 6 months old at time of enrollment - 8 courses of vincristine and carboplatin, given at 3-4 week intervals.
- Infants < 6 months old at time of enrollment - Therapy will consist of six courses of chemotherapy; three courses of vincristine and carboplatin, given at 3-4 week intervals, alternating with 3 cycles of vincristine and topotecan, given at 3-4 week intervals.
Focal treatments will be administered at the discretion of the treating team. Focal therapies will include cryotherapy, laser photocoagulation, thermo-therapy and plaque radiotherapy.
STRATUM B:
- Participants without extensive sub-retinal (SR) seeding, treatment will consist of two up-front courses of vincristine and topotecan, given at 3-4 week intervals.
- Participants without SR seeding: ≥ or equal to partial response after 2 cycles, will receive three additional courses of vincristine-topotecan (VT) and six courses of vincristine-carboplatin, given at 3-4 week intervals.
- Participants without SR seeding and < partial response after 2 cycles VT will receive 6 courses of vincristine-carboplatin-etoposide (VCE), given at 3-4 week intervals.
- Participants with extensive sub-retinal (SR) seeding will receive two up-front courses of subconjunctival (also called subtenon or periocular) CARBOplatin and systemic topotecan, given at 3-4 week intervals.
- Participants with SR seeding: > or equal to partial response after 2 cycles will receive three additional courses of vincristine-topotecan, and six courses of vincristine-carboplatin, given at 3-4 week intervals.
- Participants with SR seeding: < partial response after 2 cycles will receive 6 courses of VCE, given at 3-4 week intervals.
Focal treatments will be administered at the discretion of the treating team. Focal therapies will include cryotherapy, laser photocoagulation, thermo-therapy and plaque radiotherapy.
STRATUM C:
Participants with unilateral (unifocal or multifocal) advanced (R-E IV-V and IC D-E) intraocular disease will undergo enucleation. Adjuvant therapy will be based on histopathology:
- low risk participants: Participants in whom the enucleated eye does not show extra-retinal disease (see definition of intermediate and high risk below); will not receive any additional treatment.
- intermediate risk participants: Participants in whom the enucleated eye shows presence of tumor in the anterior chamber, invasion of the ciliary body/iris, massive invasion of the choroid, and invasion of the optic nerve beyond the lamina cribrosa with concomitant invasion of the choroid, will receive 4 courses of adjuvant chemotherapy with vincristine-carboplatin-doxorubicin (VCD).
- high risk participants: Participants in whom the enucleated eye shows involvement of the sclera, or involvement of the optic nerve at the level of the cut-end, will be treated with 6 courses of chemotherapy, with alternating courses of VCE and VCD.
- High-risk participants with extra-ocular extension (i.e. tumor extending beyond the sclera/cornea or beyond the cut end of the optic nerve) will be candidates for external-beam radiation therapy (EBRT) to the entire orbit, including the optic nerve, administered after 2 or 3 courses of treatment. Patients with extra-ocular extension may be considered for enrollment on an alternative therapeutic protocol for metastatic retinoblastoma (or best clinical management).
STRATUM D:
Management of participants with bilateral retinoblastoma is often complex; and some participants will have one eye enucleated upfront due to advanced disease. The decision for enucleation will be made after thorough consideration by the treating team. The treatment of the remaining eye will depend on a combination of two factors: a) R-E group of the remaining eye, and b) Histology of the enucleated eye. Though we have accumulated some information regarding the use of vincristine, cyclophosphamide, and doxorubicin in the treatment of intraocular retinoblastoma, it is not considered standard of care. Therefore, participants with intermediate and high risk features will be treated with 6 courses of vincristine, carboplatin, and etoposide (VCE). Those participants in whom the enucleated eye shows only low risk histology will be eligible to proceed with either stratum A or stratum B therapy. For those receiving stratum B therapy, consideration of periocular carboplatin will be allowed. External beam or proton beam radiation therapy will be considered for patients with extra-ocular extension.
Focal treatments will be administered at the discretion of the treating team. Focal therapies will include cryotherapy, laser photocoagulation, thermo-therapy and plaque radiotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Stratum A | Experimental | Drug: vincristine, Drug: topotecan, Drug: filgrastim, Drug: PEG-filgrastim, Drug: carboplatin, Other: focal therapyParticipants 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. |
| Stratum B | Experimental | Drug: vincristine, Drug: topotecan, Drug: filgrastim, Drug: PEG-filgrastim, Drug: carboplatin, Other: focal therapy, Drug: etoposideParticipants 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. |
| Stratum C | Experimental | Drug: vincristine, Drug: filgrastim, Drug: PEG-filgrastim, Drug: carboplatin, Drug: etoposide, Drug: cyclophosphamide, Drug: MESNA, Drug: doxorubicin, Procedure: enucleationParticipants with advanced (R-E IV-V and IC D-E) unilateral retinoblastoma who require upfront enucleation. Participants will be assessed and treated by low, intermediate or high risk. Interventions (see Detailed Description): vincristine, cyclophosphamide, MESNA, doxorubicin, etoposide, carboplatin, filgrastim or PEG-filgrastim, enucleation |
| Stratum D | Experimental | Drug: vincristine, Drug: topotecan, Drug: filgrastim, Drug: PEG-filgrastim, Drug: carboplatin, Other: focal therapy, Drug: etoposide, Procedure: enucleation, Radiation: external beam radiation or proton beam radiationParticipants with bilateral retinoblastoma who may require upfront enucleation for one eye due to advanced disease (R-E IV-V and IC E). Interventions (see Detailed Description): vincristine, carboplatin, topotecan, etoposide, enucleation, filgrastim or PEG-filgrastim, focal therapy, including cryotherapy, laser photocoagulation, thermotherapy (and thermo-chemotherapy) and episcleral plaque brachytherapy, and external beam radiation or proton beam radiation in select cases. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| vincristineAlso: VCR, Oncovin(R)Given via minibag/gravity flow. | Drug | Stratum A, Stratum B, Stratum C, Stratum D |
| topotecanAlso: TOPO, Hycamtin(R)Given IV over 30 minutes. | Drug | Stratum A, Stratum B, Stratum D |
| filgrastimAlso: G-CSF, Neupogen(R)Given subcutaneously 24-36 hours after chemotherapy for 7-10 days, until absolute neutrophil count (ANC) is >2,000/µL on one occasion after the expected nadir. | Drug | Stratum A, Stratum B, Stratum C, Stratum D |
| PEG-filgrastimAlso: pegylated filgrastim, PEG filgrastim, SD-01, Neulasta(R)Given subcutaneously 24-36 hours after chemotherapy for 7-10 days, until ANC is >2,000/µL on one occasion after the expected nadir. | Drug | Stratum A, Stratum B, Stratum C, Stratum D |
| carboplatinAlso: CARBO, Paraplatin(R)Given IV over 60 minutes. Given periocular (subtenon/subconjunctival). | Drug | Stratum A, Stratum B, Stratum C, Stratum D |
| focal therapyAlso: cryotherapy, laser photocoagulation, thermo-therapy, plaque radiotherapyFocal treatments will be administered at the discretion of the treating team to strata A, B and D. In select cases of very early stage retinoblastoma (Stratum A), participants may receive focal therapies only and chemotherapy will be held at the discretion of the treating team. If there is any evidence of progression or unsatisfactory results, the participant will begin chemotherapy as per Stratum A. For selected participants an effort will be made to perform sequential chemo-thermotherapy. In these cases, carboplatin will be administered one or two hours prior to thermotherapy. | Other | Stratum A, Stratum B, Stratum D |
| etoposideAlso: ETOP, VP-16, Vepesid(R), Etopophos(R)Given IV. Participants who cannot tolerate etoposide may be given etoposide phosphate (Etopophos(R)). | Drug | Stratum B, Stratum C, Stratum D |
| cyclophosphamideAlso: CYCLO, Cytoxan(R)Given IV. | Drug | Stratum C |
| MESNAAlso: Mesnex(R)Given IV before CYCLO and at 3, 6 and 9 hours after CYCLO. | Drug | Stratum C |
| doxorubicinAlso: DOXO, Adriamycin(R)Given IV on Day 1 of Cycles 2, 4 and 6 in Stratum C high-risk. | Drug | Stratum C |
| enucleationAlso: eye removalEye removal due to advanced disease in Strata C and D participants. | Procedure or surgery | Stratum C, Stratum D |
| external beam radiation or proton beam radiationAlso: EBRTEBRT or proton beam radiation will be administered to any eye in which the disease is considered to be not controllable with focal treatments alone, and in participants with enucleated eyes in which high risk of orbital and/or central nervous system disease is documented histologically (high-risk group with disease extension beyond the sclera or cornea, or beyond the cut end of the optic nerve). EBRT will be administered using standard techniques practices with the objective of limiting dose to normal tissues including the hypothalamic-pituitary unit, supratentorial brain, orbit, cochleae and contralateral eye when indicated. Participants will be evaluated on an individual basis to determine whether they might benefit from referral for proton therapy. | Radiation | Stratum D |
Primary outcome
Response Rate
Time frame: Through completion of two upfront courses of therapy (each course lasting 3-4 weeks; total approximately 6-8 weeks)
To evaluate the response (CR + PR) rate of bilateral disease patients who have at least one eye with advanced intraocular retinoblastoma (stratum B patients) and subretinal seeding to two upfront courses of therapy consisting of subconjunctival carboplatin and systemic topotecan. Stratum B patients who had no significant subretinal seeding and received vincristine and topotecan are not evaluable for this primary objective. CR: Complete regression, calcification or scarring of all apparent tumor masses in the funduscopic examination. Lesions which initially decrease in size and then remain unchanged (but incompletely calcified or scarred) in the absence of further focal or systemic therapy will also be considered CR. The response must persist for at least 4 weeks. PR: Greater than 50% (but less than 100%) reduction of the tumor masses in the funduscopic examination, without the appearance of any new lesions. The response must persist for at least 4 weeks.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- St. Jude Children's Research HospitalLink provided by the sponsor.
- Clinical Trials Open at St. JudeLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carlos Rodriguez-Galindo, MD principal investigator
St. Jude Children's Research Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing vincristine, topotecan and filgrastim in people with Retinoblastoma, run by St. Jude Children's Research Hospital at 1 site in United States.
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 5 inclusion and 4 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: under way, not looking for participants, as of the sponsor's last update on 29 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Response Rate (time frame: Through completion of two upfront courses of therapy (each course lasting 3-4 weeks; total approximately 6-8 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Jun 2026; AzTrials last refreshed its copy on 7 Nov 2026.