MedPath

Studying Health Outcomes After Treatment in Patients With Retinoblastoma

Recruiting
Conditions
Cancer Survivor
Biological Sibling
Retinoblastoma
Intraocular Retinoblastoma
Unilateral Retinoblastoma
Interventions
Procedure: Biospecimen collection
Other: Vision assessment
Other: Questionnaire administration
Other: Quality of life assessment
Other: Laboratory Biomarker Analysis
Registration Number
NCT03932786
Lead Sponsor
Vanderbilt-Ingram Cancer Center
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

Detailed Description

PRIMARY OBJECTIVES:

I. 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.

II. 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.

III. 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.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
900
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
Prospective (biospecimens, vision assessment, questionnaires)Biospecimen collection-
Prospective (biospecimens, vision assessment, questionnaires)Questionnaire administration-
Retrospective(biospecimens, vision assessment, questionnaires)Biospecimen collection-
Retrospective(biospecimens, vision assessment, questionnaires)Questionnaire administration-
Retrospective(biospecimens, vision assessment, questionnaires)Quality of life assessment-
Retrospective(biospecimens, vision assessment, questionnaires)Vision assessment-
Retrospective(biospecimens, vision assessment, questionnaires)Laboratory Biomarker Analysis-
Prospective (biospecimens, vision assessment, questionnaires)Quality of life assessment-
Prospective (biospecimens, vision assessment, questionnaires)Vision assessment-
Prospective (biospecimens, vision assessment, questionnaires)Laboratory Biomarker Analysis-
Primary Outcome Measures
NameTimeMethod
Assess quality of life utilizing questionnaires: CBCLUp to 2 years
Assess psycho-social outcomes utilizing questionnaires: BRIEF-P,Up to 2 years
Estimate malignant neoplasm (SMN) risk .Measured through medical record abstraction,Up to 1 year
Assess visual outcomes measured via age appropriate visual acuity testingUp to 1 year
Incidence of acute toxicityUp to 1 year
Assess psycho-social outcomes utilizing questionnaires: BRIEFUp 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: Pediatric Quality of LifeUp to 2 years
Assess visual outcomes measured via parent reportUp to 1 year
Assess quality of life utilizing questionnaires: BRIEFUp to 2 years
Assess psycho-social outcomes utilizing questionnaires: CBCLUp to 2 years
Assess quality of life utilizing questionnaires: Youth Self-ReportUp to 2 years
Assess psycho-social outcomes utilizing questionnaires: Youth Self-ReportUp to 2 years
Assess psycho-social outcomes utilizing questionnaires: Pediatric Quality of LifeUp to 2 years
Assess visual outcomes measured via vision questionnairesUp to 1 year
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (11)

University of Minnesoa

🇺🇸

Minneapolis, Minnesota, United States

Texas Childeren's Hospital

🇺🇸

Houston, Texas, United States

Washington School of Medicine at St. Louis

🇺🇸

Saint Louis, Missouri, United States

Lurie Children's Hospital

🇺🇸

Chicago, Illinois, United States

The Hosptial for Sick Children

🇨🇦

Toronto, Canada

Vanderbilt-Ingram Cancer Center

🇺🇸

Nashville, Tennessee, United States

University of Illinois, Chicago

🇺🇸

Chicago, Illinois, United States

Children's Hospital of Philadelphia

🇺🇸

Philadelphia, Pennsylvania, United States

Cincinnati Children's Hospital Medical Center

🇺🇸

Cincinnati, Ohio, United States

Children's Hospital of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

© Copyright 2025. All Rights Reserved by MedPath