RIVERBOAT: Studying Health Outcomes After Treatment in Patients With Retinoblastoma
Study Details
Study Description
Brief Summary
This trial studies health outcomes after treatment in patients with retinoblastoma. Gathering health information over time from patients and family members through vision assessments, samples of tissue and saliva, and questionnaires may help doctors learn more about what causes retinoblastoma, identify long-term health outcomes for patients with retinoblastoma, and find out which therapies may be the best for treating retinoblastoma
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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Detailed Description
PRIMARY OBJECTIVES:
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Define acute toxicity, subsequent malignant neoplasm (SMN) risk and visual outcomes in retinoblastoma (RB) survivors and compare patient centered psychosocial and neurocognitive and physical outcomes in survivors with normative data and sibling controls.
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Create the first Clinically-Annotated Patient Tissues to Analyze Gene INteractions to assess biologic correlates of disease and facilitate future research: The RIVERBOAT-CAPTAIN biorepository, including germline deoxyribonucleic acid (DNA) and tumor tissue from patients, with detailed patient, disease and treatment-related information.
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Using the RIVERBOAT-CAPTAIN clinically-annotated biorepository, determine the interplay between specific RB1 mutation type and the role of additional modifier genes in determining those tumor phenotypes that drive treatment decisions.
OUTLINE: Patients are assigned to 1 of 2 cohorts.
RETROSPECTIVE COHORT: Patients treated between 2008-2018 undergo collection of saliva samples at >= 6 months after treatment, and undergo vision assessment at >= 6 months after treatment and again 1 year later if necessary. Previously collected tissue samples at the time of surgery are also obtained. Patients also complete questionnaires at >= 6 months after treatment and again 2 years later.
PROSPECTIVE COHORT: Patients treated between 2018-2023 undergo collection of saliva samples at the time of enrollment and at 6 months after treatment. Patients also undergo vision assessment at the time of enrollment, at 6 months, and 18 months after completion of treatment. Patients also complete questionnaires at 6 months and again 2 years later, as well as undergo collection of tissue samples at the time of surgery. Immediate family members with history of RB or RB1 gene mutation also undergo collection saliva samples.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Retrospective(biospecimens, vision assessment, questionnaires)
|
Procedure: Biospecimen collection
Collection of tissue and saliva samples
Other: Vision assessment
Undergo vision assessment
Other: Questionnaire administration
Complete questionnaires
Other: Quality of life assessment
Complete questionnaires
Other: Laboratory Biomarker Analysis
Correlative studies
|
| Prospective (biospecimens, vision assessment, questionnaires)
|
Procedure: Biospecimen collection
Collection of tissue and saliva samples
Other: Vision assessment
Undergo vision assessment
Other: Questionnaire administration
Complete questionnaires
Other: Quality of life assessment
Complete questionnaires
Other: Laboratory Biomarker Analysis
Correlative studies
|
Outcome Measures
Primary Outcome Measures
- Incidence of acute toxicity [Up to 1 year]
- Estimate malignant neoplasm (SMN) risk .Measured through medical record abstraction, [Up to 1 year]
- Assess visual outcomes measured via age appropriate visual acuity testing [Up to 1 year]
- Assess psycho-social outcomes utilizing questionnaires: BRIEF [Up to 2 years]
- Genes will be tested to examine the role they play in Retinoblastoma. This will be done via whole-exome sequencing and whole RB1 Gene examination. [Up to 1 year]
- Assess quality of life utilizing questionnaires: BRIEF [Up to 2 years]
- Assess quality of life utilizing questionnaires: CBCL [Up to 2 years]
- Assess quality of life utilizing questionnaires: Youth Self-Report [Up to 2 years]
- Assess quality of life utilizing questionnaires: Pediatric Quality of Life [Up to 2 years]
- Assess visual outcomes measured via parent report [Up to 1 year]
- Assess visual outcomes measured via vision questionnaires [Up to 1 year]
- Assess psycho-social outcomes utilizing questionnaires: BRIEF-P, [Up to 2 years]
- Assess psycho-social outcomes utilizing questionnaires: CBCL [Up to 2 years]
- Assess psycho-social outcomes utilizing questionnaires: Youth Self-Report [Up to 2 years]
- Assess psycho-social outcomes utilizing questionnaires: Pediatric Quality of Life [Up to 2 years]
Eligibility Criteria
Criteria
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Unilateral or bilateral intraocular retinoblastoma
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Diagnosis between the ages of 0 - 17.99 years
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Diagnosis on or after January 1, 2008
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No exclusions based on primary or secondary treatment modalities
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Retrospective group patients must be ≥ 6 months post end of treatment at study entry
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For those already at this timepoint, they are now eligible
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For those in treatment, or otherwise not yet at this timepoint, they are eligible once at they are ≥ 6 months post end of treatment
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Prospective group patients must not have begun treatment
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Patients with diminished capacity will not be enrolled.
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Language: Patients must be able to communicate in English, French, or Spanish
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Sibling Cohort: One sibling, not affected by retinoblastoma will be enrolled, preference for the sibling closest in age to the RB patient.
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Regulatory Requirements: All patients and/or their parents or legal guardians must sign a written informed consent. All institutional, FDA, and NCI requirements for human studies must be met.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Lurie Children's Hospital | Chicago | Illinois | United States | 60611 |
| 2 | University of Illinois, Chicago | Chicago | Illinois | United States | 60612 |
| 3 | University of Minnesoa | Minneapolis | Minnesota | United States | 55455 |
| 4 | Washington School of Medicine at St. Louis | Saint Louis | Missouri | United States | 63110 |
| 5 | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | United States | 45229 |
| 6 | Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | United States | 19104 |
| 7 | Vanderbilt-Ingram Cancer Center | Nashville | Tennessee | United States | 37232 |
| 8 | MD Anderson Cancer Center | Houston | Texas | United States | 77030 |
| 9 | Texas Childeren's Hospital | Houston | Texas | United States | 77030 |
| 10 | Children's Hospital of Wisconsin | Milwaukee | Wisconsin | United States | 53226 |
| 11 | The Hosptial for Sick Children | Toronto | Canada |
Sponsors and Collaborators
- Vanderbilt-Ingram Cancer Center
- National Cancer Institute (NCI)
Investigators
- Principal Investigator: Debra Friedman, MD, Vanderbilt Medical Center
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- VICC PED 1878
- NCI-2019-00635
- 1R01CA225005-01A1