Trial · NCT00186888
Study of Treatment for Patients With Cancer of the Eye -Retinoblastoma
NCT00186888, Phase 3, Completed, St. Jude Children's Research Hospital; results first posted 2011-08-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Treatment for Patients With Cancer of the Eye -Retinoblastoma. Trials, record NCT00186888. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00186888
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Treatment for Patients With Cancer of the Eye -Retinoblastoma
- NCT number
- NCT00186888
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 107 participants, actual
- Lead sponsor
- St. Jude Children's Research Hospital
- Conditions
- Retinoblastoma, Retinal Neoplasm
- Interventions
Enucleation, Vincristine, Carboplatin, Focal Therapies, External Beam Radiation, Vincristine and Topotecan, Vincristine + Carboplatin + Etoposide, vincristine, cyclophosphamide, and doxorubicin, Vincristine, Carboplatin and Etoposide, Periocular carboplatin, G-CSF
- First posted
- 2005-09-16
- Last updated
- 2025-09-04
- Results first posted
- 2011-08-30
Source ClinicalTrials.gov
Registered outcomes30
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Stratum B Response to Window Therapy | Six weeks post window therapy | The primary outcome is to estimate the proportion of stratum B patients responding to 2 courses of window therapy consisting of vincristine and topotecan. Complete Response is the complete regression of all apparent tumor masses in the funduscopic examination and by MRI and ultrasound (US). Partial Response is defined as greater than 50% (but less than 100%) reduction of the tumor masses in the funduscopic examination and by US and MRI, without the appearance of any new lesions. The response must persist for at least 4 weeks. Stratum A and C did not receive window therapy. |
| Secondary | Stratum B Response Rate of Early Stage Eyes to Window Therapy | Six weeks post window therapy. | To estimate the proportion of early stage eyes defined as Reese-Ellsworth Group I, II, or III eyes, that responded to 2 courses of window therapy which consisted of vincristine and topotecan |
| Secondary | Relationship Between Topotecan Clearance (CL) and CYP3A4/5 Genotype in Stratum B Participants. | Courses 1, 2, 5, and 8 | Blood samples for pharmacokinetic studies were collected at 0 hour (pre-dose), 5 minutes, 1.5 and 2.5 hours after the end of topotecan dose on Course 1 Day 1, Course 2 Day 1, and if further studies were needed, Course 5 Day 1 and Course 8 Day 1. A blood sample for pharmacogenetic studies was collected during the course of therapy on protocol. |
| Secondary | Relationship Between Topotecan Clearance (CL) and ABCG2/B1 Genotype in Stratum B Participants. | Courses 1, 2, 5, and 8 | Blood samples for pharmacokinetic studies were collected at 0 hour (pre-dose), 5 minutes, 1.5 and 2.5 hours after the end of topotecan dose on Course 1 Day 1, Course 2 Day 1, and if further studies were needed, Course 5 Day 1 and Course 8 Day 1. A blood sample for pharmacogenetic studies was collected during the course of therapy on protocol. |
| Secondary | Event-free Survival of Stratum B Patients Responding to Window Treatment | From date on-study to an event or last follow-up | To estimate the 5-year event-free (EFS) survival of bilateral disease patients with advanced intraocular retinoblastoma in either eye (R-E IV-V) responding to the vincristine/topotecan window, with alternating cycles of vincristine and carboplatin with vincristine, topotecan, and periocular carboplatin, with intensive focal treatments. Event-free survival will be defined per patient as follows: for patients with one advanced stage eye, the time interval from date on study to date of first event (an event includes external beam radiation or enucleation) of advanced stage eyes, time to first event will be used for the analysis. Event-free survival will be estimated using the method of Kaplan and Meier. |
| Secondary | Ocular Survival of Stratum B Patients Responding to Window Treatment | From date on-study to an event or last follow-up | To estimate the 5-year ocular survival of bilateral disease patients with advanced intraocular retinoblastoma in either eye (R-E IV-V) responding to the vincristine/topotecan window, with alternating cycles of vincristine and carboplatin with vincristine, topotecan, and periocular carboplatin, with intensive focal treatments. Ocular survival will be defined per patient as follows: for patients with one advanced stage eye, the time interval from date on study to date of enucleation of advanced stage eye or date of last follow-up, for patients with two advanced stage eyes, the time to the first enucleation will be used for analysis. Ocular survival will be estimated using the method of Kaplan and Meier. Standard error is 5-year ocular survival |
| Secondary | Event-free Survival of Eyes in Stratum B Patients Responding to Window Treatment | From date on-study to an event or last follow-up | To estimate the 5-year event-free survival (EFS) of eyes of bilateral disease patients with advanced intraocular retinoblastoma in either eye (R-E IV-V) responding to the vincristine/topotecan window, with alternating cycles of vincristine and carboplatin with vincristine, topotecan, and periocular carboplatin, with intensive focal treatments. Event-free survival of eye will be defined per eye as the time interval from date on study to date of first event (an event includes external beam radiation or enucleation) or to last follow-up date for eyes without events. Event-free survival of eye will be estimated using the method of Kaplan and Meier. Standard error is 5-year EFS. |
| Secondary | Ocular Survival of Eyes in Stratum B Patients Responding to Window Treatment | From date on-study to an event or last follow-up | To estimate the 5-year ocular survival of eye of bilateral disease patients with advanced intraocular retinoblastoma in either eye (R-E IV-V) responding to the vincristine/topotecan window, with alternating cycles of vincristine and carboplatin with vincristine, topotecan, and periocular carboplatin, with intensive focal treatments. Ocular survival of eye will be defined per eye as the time interval from date on study to date of enucleation or date of last follow-up. Ocular survival of eye will be estimated using the method of Kaplan and Meier. Standard error is 5-year ocular survival. |
| Secondary | Event-free Survival of Stratum B Patients Not Responding to Window Treatment | From date on-study to an event or last follow-up | To estimate the 5-year event free survival of patients with advanced intraocular retinoblastoma (R-E IV-V) not responding to the vincristine/topotecan window, with a combination of vincristine, carboplatin, etoposide, and periocular carboplatin, with intensive focal treatments. |
| Secondary | Ocular Survival of Stratum B Patients Not Responding to Window Treatment | From date on-study to an event or last follow-up | To estimate the 5-year ocular survival of patients with advanced intraocular retinoblastoma (R-E IV-V) not responding to the vincristine/topotecan window, with a combination of vincristine, carboplatin, etoposide, and periocular carboplatin, with intensive focal treatments. |
1 to 10 of 30Source ClinicalTrials.gov
See all 30 registered outcomesOutcomes31
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Stratum B Response to Window Therapy | Six weeks post window therapy | The primary objective related to stratum B patients only, as these were the patients who were given window therapy consisting of 2 courses of vincristine and topotecan. Of the 27 stratum B patients enrolled, all were included in the analysis of the primary objective. | Participants | NUMBER |
| SECONDARY | Stratum B Response Rate of Early Stage Eyes to Window Therapy | Six weeks post window therapy. | Among the 27 stratum B patients with 54 eyes with retinoblastoma, 12 eyes were early stage (Reese-Ellsworth group I, II, or III). The remaining 42 eyes were advanced stage and were not included in this analysis. | Participants | NUMBER |
| SECONDARY | Relationship Between Topotecan Clearance (CL) and CYP3A4/5 Genotype in Stratum B Participants. | Courses 1, 2, 5, and 8 | Of the 107 participants enrolled in the overall study, analysis was performed for 19 participants who were enrolled on Stratum B AND who had results for both topotecan clearance and pharmacogenetic studies.~Only wild-type was present in CYP3A5\*6, therefore, statistical analysis was not done for these alleles. | Liters/hour/m^2 | MEDIAN |
| SECONDARY | Relationship Between Topotecan Clearance (CL) and ABCG2/B1 Genotype in Stratum B Participants. | Courses 1, 2, 5, and 8 | Of the 107 participants enrolled in the overall study, analysis was performed for 19 participants who were enrolled on Stratum B AND who had results for both topotecan clearance and pharmacogenetic studies.~Only wild-type was present in BCRP 15994, therefore, statistical analysis was not done for these alleles. | Liters/hour/m^2 | MEDIAN |
| SECONDARY | Event-free Survival of Stratum B Patients Responding to Window Treatment | From date on-study to an event or last follow-up | Of the total 27 eligible patients in stratum B, 3 patients were not responding to the window therapy; 1 withdrew the consent and was taken off the study, and 2 developed disease progression. Kaplan and Meier estimate of ocular survival was calculated for the remaining 24 patients. | probability | NUMBER |
| SECONDARY | Ocular Survival of Stratum B Patients Responding to Window Treatment | From date on-study to an event or last follow-up | From the total of 27 eligible patients in stratum B, 3 patients were not responding to the window therapy; 1 withdrew the consent and was taken off the study, and 2 developed disease progression. Thus, the Kaplan and Meier estimate of ocular survival was calculated for the remaining 24 patients. | probability | NUMBER |
| SECONDARY | Event-free Survival of Eyes in Stratum B Patients Responding to Window Treatment | From date on-study to an event or last follow-up | Of the 52 eyes (26 evaluable patients), 2 eyes (2 patients) were removed from analysis as they were not responsive to window therapy. In both cases, the contralateral eye was included in the analysis. One patient with upfront enucleation had only one eye for analysis. Eleven eyes were R-E Group I-III and were excluded. Total: 38 eyes for analysis. | probability | NUMBER |
| SECONDARY | Ocular Survival of Eyes in Stratum B Patients Responding to Window Treatment | From date on-study to an event or last follow-up | Of the 52 eyes (26 evaluable patients), 2 eyes (2 patients) were removed from analysis as they were not responsive to window therapy. In both cases, the contralateral eye was included in the analysis. One patient with upfront enucleation had only one eye for analysis. Eleven eyes were R-E Group I-III and were excluded. Total: 38 eyes for analysis. | probability | NUMBER |
| SECONDARY | Event-free Survival of Stratum B Patients Not Responding to Window Treatment | From date on-study to an event or last follow-up | The study closed to enrollment early due to poor accrual, but it remains open to follow-up. Both patients who developed new lesions in one eye during window therapy had a good response in the contralateral eye, and they continued on protocol therapy with vincristine/topotecan. Therefore, no patients were treated with this combination therapy. | ||
| SECONDARY | Ocular Survival of Stratum B Patients Not Responding to Window Treatment | From date on-study to an event or last follow-up | The study closed to enrollment early due to poor accrual, but it remains open to follow-up. Both patients who developed new lesions in one eye during window therapy had a good response in the contralateral eye, and they continued on protocol therapy with vincristine/topotecan. Therefore, no patients were treated with this combination therapy. |
1 to 10 of 31Source ClinicalTrials.gov
See all 31 outcomesResults by group108
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Stratum B Response to Window Therapy | Stratum B | Partial response | 24 | Participants | |
| Stratum B Response to Window Therapy | Stratum B | Progressive Disease or New Lesion | 2 | Participants | |
| Stratum B Response to Window Therapy | Stratum B | Failure due to Toxicity | 1 | Participants | |
| Stratum B Response Rate of Early Stage Eyes to Window Therapy | Stratum B | Partial response | 11 | Participants | |
| Stratum B Response Rate of Early Stage Eyes to Window Therapy | Stratum B | Progressive Disease / New lesion | 0 | Participants | |
| Stratum B Response Rate of Early Stage Eyes to Window Therapy | Stratum B | Failure due to Toxicity | 1 | Participants | |
| Relationship Between Topotecan Clearance (CL) and CYP3A4/5 Genotype in Stratum B Participants. | Stratum B | 18.8 | 95% Confidence Interval 9.8 to 36.8 | Liters/hour/m^2 | |
| Relationship Between Topotecan Clearance (CL) and ABCG2/B1 Genotype in Stratum B Participants. | Stratum B | 18.8 | 95% Confidence Interval 9.8 to 36.8 | Liters/hour/m^2 | |
| Event-free Survival of Stratum B Patients Responding to Window Treatment | Stratum B | 0.667 | 95% Confidence Interval 0.478 to 0.855 | probability | |
| Ocular Survival of Stratum B Patients Responding to Window Treatment | Stratum B | 0.667 | 95% Confidence Interval 0.478 to 0.855 | probability |
1 to 10 of 108Source ClinicalTrials.gov
See all 108 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Stratum A | 23 | |
| Overall Study | STARTED | Stratum B | 27 | |
| Overall Study | STARTED | Stratum C | 55 | |
| Overall Study | Window Therapy | Stratum A | 0 | |
| Overall Study | Window Therapy | Stratum B | 27 | |
| Overall Study | Window Therapy | Stratum C | 0 | |
| Overall Study | COMPLETED | Stratum A | 19 | |
| Overall Study | COMPLETED | Stratum B | 24 | |
| Overall Study | COMPLETED | Stratum C | 55 | |
| Overall Study | NOT COMPLETED | Stratum A | 4 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events87
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Neutrophils/granulocytes (ANC/AGC) | Blood and lymphatic system disorders | Stratum A | 23 | 23 | 161 |
| other | Neutrophils/granulocytes (ANC/AGC) | Blood and lymphatic system disorders | Stratum B | 26 | 27 | 271 |
| other | Neutrophils/granulocytes (ANC/AGC) | Blood and lymphatic system disorders | Stratum C | 18 | 55 | 69 |
| other | Platelets | Blood and lymphatic system disorders | Stratum A | 9 | 23 | 22 |
| other | Platelets | Blood and lymphatic system disorders | Stratum B | 25 | 27 | 86 |
| other | Platelets | Blood and lymphatic system disorders | Stratum C | 10 | 55 | 32 |
| other | Hemoglobin | Blood and lymphatic system disorders | Stratum A | 10 | 23 | 22 |
| other | Hemoglobin | Blood and lymphatic system disorders | Stratum B | 23 | 27 | 72 |
| other | Hemoglobin | Blood and lymphatic system disorders | Stratum C | 14 | 55 | 34 |
| other | Leukocytes | Blood and lymphatic system disorders | Stratum A | 3 | 23 | 5 |
1 to 10 of 87Source ClinicalTrials.gov
See all 87 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Stratum A | ||
| Total, all-cause mortality | Stratum B | ||
| Total, all-cause mortality | Stratum C | ||
| Total, other adverse events | Stratum A | 23 | 23 |
| Total, other adverse events | Stratum B | 27 | 27 |
| Total, other adverse events | Stratum C | 18 | 55 |
| Total, serious adverse events | Stratum A | 1 | 23 |
| Total, serious adverse events | Stratum B | 9 | 27 |
| Total, serious adverse events | Stratum C | 1 | 55 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 33507604 | DERIVED |
Source PubMed
Grants1
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| P01CA023099 | Studies of Childhood Solid Tumors | HENDERSHOT, LINDA M | ST. JUDE CHILDREN'S RESEARCH HOSPITAL | NATIONAL CANCER INSTITUTE | 1985 to 2011 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00186888
GET https://conductscience.org/api/trials/v1/NCT00186888/registered?page=2