Trial Outcomes & Findings for Protocol for the Study and Treatment of Participants With Intraocular Retinoblastoma (NCT NCT01783535)

Last Updated: 2026-06-29

Results Overview

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.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

174 participants

Primary outcome timeframe

Through completion of two upfront courses of therapy (each course lasting 3-4 weeks; total approximately 6-8 weeks)

Results posted on

2026-06-29

Participant Flow

174 total patients were recruited at 1 SJCRH. One patient was removed from the protocol without treatment; therefore, the report is based on 173 patients. Stratum A has 31 patients recruited between October 4, 2013 and August 4, 2022. Stratum B has 46 patients recruited and between January 31, 2014 and February 21, 2023. Stratum C has 83 patients recruited between June 19, 2013 and December 6, 2022. Stratum D has 13 patients recruited between October 8, 2013 and February 11, 2021.

174 total patients were enrolled on the study. One patient was removed from the protocol without treatment; therefore, the report is based on the rest of the 173 patients.

Participant milestones

Participant milestones
Measure	Stratum A Stratum A has 31 patients recruited between October 4, 2013 and August 4, 2022. Stratum A: Early Unilateral or Bilateral Retinoblastoma. 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.	Stratum B Stratum B has 46 patients recruited and between January 31, 2014 and February 21, 2023. Stratum B: Advanced Bilateral Retinoblastoma or Advanced Unilateral with Foveal Sparing Retinoblastoma. Participants who are considered candidates for conservative management including: Participants with bilateral retinoblastoma who have R-E IV-V and/or IC D-E in one eye.	Stratum C Stratum C has 83 patients recruited between June 19, 2013 and December 6, 2022. Stratum C: Advanced Unilateral Retinoblastoma. Participants with advanced (R-E IV-V and/or IC D-E) unilateral retinoblastoma who require upfront enucleation.	Stratum D Stratum D has 13 patients recruited between October 8, 2013 and February 11, 2021. Stratum D: Bilateral Retinoblastoma with Upfront Enucleation of One Eye due to Advanced Disease. Participants with bilateral retinoblastoma who may require upfront enucleation for one eye due to advanced disease (R-E IV-V and/or IC E).
Patients That Completed Protocol Therapy STARTED	31	46	83	13
Patients That Completed Protocol Therapy COMPLETED	29	44	80	11
Patients That Completed Protocol Therapy NOT COMPLETED	2	2	3	2
Patients That Completed Overall Study STARTED	31	46	83	13
Patients That Completed Overall Study COMPLETED	2	2	6	1
Patients That Completed Overall Study NOT COMPLETED	29	44	77	12

Reasons for withdrawal

Reasons for withdrawal
Measure	Stratum A Stratum A has 31 patients recruited between October 4, 2013 and August 4, 2022. Stratum A: Early Unilateral or Bilateral Retinoblastoma. 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.	Stratum B Stratum B has 46 patients recruited and between January 31, 2014 and February 21, 2023. Stratum B: Advanced Bilateral Retinoblastoma or Advanced Unilateral with Foveal Sparing Retinoblastoma. Participants who are considered candidates for conservative management including: Participants with bilateral retinoblastoma who have R-E IV-V and/or IC D-E in one eye.	Stratum C Stratum C has 83 patients recruited between June 19, 2013 and December 6, 2022. Stratum C: Advanced Unilateral Retinoblastoma. Participants with advanced (R-E IV-V and/or IC D-E) unilateral retinoblastoma who require upfront enucleation.	Stratum D Stratum D has 13 patients recruited between October 8, 2013 and February 11, 2021. Stratum D: Bilateral Retinoblastoma with Upfront Enucleation of One Eye due to Advanced Disease. Participants with bilateral retinoblastoma who may require upfront enucleation for one eye due to advanced disease (R-E IV-V and/or IC E).
Patients That Completed Protocol Therapy Physician Decision	1	2	1	2
Patients That Completed Protocol Therapy Progressive disease	0	0	2	0
Patients That Completed Protocol Therapy Missing Pending	1	0	0	0
Patients That Completed Overall Study Physician Decision	3	14	4	5
Patients That Completed Overall Study Lost to Follow-up	0	0	1	0
Patients That Completed Overall Study Missing Pending	26	29	72	7
Patients That Completed Overall Study Missing Not Applicable	0	1	0	0

Baseline Characteristics

Protocol for the Study and Treatment of Participants With Intraocular Retinoblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Stratum A n=31 Participants Early Unilateral or Bilateral Retinoblastoma	Stratum B n=46 Participants Advanced Bilateral Retinoblastoma or Advanced Unilateral with Foveal Sparing Retinoblastoma.	Stratum C n=83 Participants Advanced Unilateral Retinoblastoma	Stratum D n=13 Participants Bilateral Retinoblastoma with Upfront Enucleation of One Eye due to Advanced Disease.	Total n=173 Participants Total of all reporting groups
Age, Continuous	14.8 Months STANDARD_DEVIATION 22.9 • n=9 Participants	15.6 Months STANDARD_DEVIATION 12.5 • n=27 Participants	27.6 Months STANDARD_DEVIATION 19.7 • n=267 Participants	19.2 Months STANDARD_DEVIATION 32.5 • n=265 Participants	21.5 Months STANDARD_DEVIATION 20.7 • n=568 Participants
Sex: Female, Male Female	18 Participants n=9 Participants	16 Participants n=27 Participants	36 Participants n=267 Participants	2 Participants n=265 Participants	72 Participants n=568 Participants
Sex: Female, Male Male	13 Participants n=9 Participants	30 Participants n=27 Participants	47 Participants n=267 Participants	11 Participants n=265 Participants	101 Participants n=568 Participants
Race/Ethnicity, Customized American Ind / Alaskan / Black	0 Participants n=9 Participants	1 Participants n=27 Participants	0 Participants n=267 Participants	0 Participants n=265 Participants	1 Participants n=568 Participants
Race/Ethnicity, Customized American Ind / Alaskan / White	0 Participants n=9 Participants	1 Participants n=27 Participants	0 Participants n=267 Participants	0 Participants n=265 Participants	1 Participants n=568 Participants
Race/Ethnicity, Customized Asian	0 Participants n=9 Participants	0 Participants n=27 Participants	2 Participants n=267 Participants	0 Participants n=265 Participants	2 Participants n=568 Participants
Race/Ethnicity, Customized Asian and White	1 Participants n=9 Participants	1 Participants n=27 Participants	0 Participants n=267 Participants	0 Participants n=265 Participants	2 Participants n=568 Participants
Race/Ethnicity, Customized Black	5 Participants n=9 Participants	8 Participants n=27 Participants	28 Participants n=267 Participants	5 Participants n=265 Participants	46 Participants n=568 Participants
Race/Ethnicity, Customized Black and White	2 Participants n=9 Participants	1 Participants n=27 Participants	3 Participants n=267 Participants	0 Participants n=265 Participants	6 Participants n=568 Participants
Race/Ethnicity, Customized Multiple Race (NOS)	0 Participants n=9 Participants	2 Participants n=27 Participants	3 Participants n=267 Participants	0 Participants n=265 Participants	5 Participants n=568 Participants
Race/Ethnicity, Customized White	23 Participants n=9 Participants	32 Participants n=27 Participants	47 Participants n=267 Participants	8 Participants n=265 Participants	110 Participants n=568 Participants
Primary diagnosis at time of enrollment Retinoblastoma, Eye, Bilateral	12 Participants n=9 Participants	35 Participants n=27 Participants	0 Participants n=267 Participants	12 Participants n=265 Participants	59 Participants n=568 Participants
Primary diagnosis at time of enrollment Retinoblastoma, Eye, Bilateral (Asynchronous)	0 Participants n=9 Participants	0 Participants n=27 Participants	3 Participants n=267 Participants	0 Participants n=265 Participants	3 Participants n=568 Participants
Primary diagnosis at time of enrollment Retinoblastoma, Eye, Left	10 Participants n=9 Participants	4 Participants n=27 Participants	48 Participants n=267 Participants	0 Participants n=265 Participants	62 Participants n=568 Participants
Primary diagnosis at time of enrollment Retinoblastoma, Eye, Right	9 Participants n=9 Participants	7 Participants n=27 Participants	32 Participants n=267 Participants	0 Participants n=265 Participants	48 Participants n=568 Participants
Primary diagnosis at time of enrollment Retinoblastoma, Multifocal, Eye, Bilateral	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants	1 Participants n=265 Participants	1 Participants n=568 Participants
Age	8.2 Months n=9 Participants	13.0 Months n=27 Participants	24.0 Months n=267 Participants	13.0 Months n=265 Participants	17.0 Months n=568 Participants

PRIMARY outcome

Timeframe: Through completion of two upfront courses of therapy (each course lasting 3-4 weeks; total approximately 6-8 weeks)

Outcome measures

Outcome measures
Measure	Stratum B With Seeding n=30 Participants Stratum B: Advanced Bilateral Retinoblastoma or Advanced Unilateral with Foveal Sparing Retinoblastoma. Participants who are considered candidates for conservative management including: Participants with bilateral retinoblastoma who have R-E IV-V and/or IC D-E in one eye. Stratum B patients who had no significant subretinal seeding and received vincristine and topotecan are not evaluable for this primary objective. Participants who have complete or partial response to two upfront courses of therapy are counted as responders. In this study we have 30 responders out of the total 30 stratum B patients with seeding.
Response Rate	30 Participants

Adverse Events

Stratum A

Serious events: 0 serious events

Other events: 25 other events

Deaths: 0 deaths

Stratum B

Serious events: 1 serious events

Other events: 45 other events

Deaths: 0 deaths

Stratum C

Serious events: 0 serious events

Other events: 24 other events

Deaths: 0 deaths

Stratum D

Serious events: 0 serious events

Other events: 7 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Stratum A n=31 participants at risk Early Unilateral or Bilateral Retinoblastoma	Stratum B n=46 participants at risk Advanced Bilateral Retinoblastoma or Advanced Unilateral with Foveal Sparing Retinoblastoma	Stratum C n=83 participants at risk Advanced Unilateral Retinoblastoma	Stratum D n=13 participants at risk Bilateral Retinoblastoma with Upfront Enucleation of One Eye due to Advanced Disease.
Investigations Neutrophil count decreased	0.00% 0/31 • Non-serious Grade 3-4 adverse events were collected during protocol therapy (up to about 19 months per patient). All-cause mortality and serious adverse events were collected through the March 19, 2024 data cutoff (up to about 10.7 years per patient). CTCAE v4.0 Grades 3-4 toxicities	2.2% 1/46 • Number of events 1 • Non-serious Grade 3-4 adverse events were collected during protocol therapy (up to about 19 months per patient). All-cause mortality and serious adverse events were collected through the March 19, 2024 data cutoff (up to about 10.7 years per patient). CTCAE v4.0 Grades 3-4 toxicities	0.00% 0/83 • Non-serious Grade 3-4 adverse events were collected during protocol therapy (up to about 19 months per patient). All-cause mortality and serious adverse events were collected through the March 19, 2024 data cutoff (up to about 10.7 years per patient). CTCAE v4.0 Grades 3-4 toxicities	0.00% 0/13 • Non-serious Grade 3-4 adverse events were collected during protocol therapy (up to about 19 months per patient). All-cause mortality and serious adverse events were collected through the March 19, 2024 data cutoff (up to about 10.7 years per patient). CTCAE v4.0 Grades 3-4 toxicities

Other adverse events

Additional Information

CARLOS RODRIGUEZ-GALINDO, MD

St. Jude Children's Research Hospital

Phone: 866-278-5833

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place