Studying Health Outcomes After Treatment in Patients With Retinoblastoma

Cancer Survivor Clinical Trial

— RIVERBOAT  

Official title:

Research Into Visual Endpoints and RB Health Outcomes After Treatment (RIVERBOAT)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03932786
• Other study ID #: VICC PED 1878
• Secondary ID: NCI-2019-006351R
• Status: Recruiting
• Start date: January 24, 2019
• Est. completion date: January 2031

Study information

• Verified date: May 2024
• Source: Vanderbilt-Ingram Cancer Center
• Contact: Vanderbilt-Ingram Service for Timely Access
• Phone: 800-811-8480
• Email: cip[@]vanderbilt.edu
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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

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 information / eligibility

• Status: Recruiting
• Enrollment: 900
• Est. completion date: January 2031
• Est. primary completion date: January 2030
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: N/A and older

Eligibility

• Unilateral or bilateral intraocular retinoblastoma
• Diagnosis between the ages of 0 - 17.99 years
• Diagnosis on or after January 1, 2008
• No exclusions based on primary or secondary treatment modalities
• Retrospective group patients must be = 6 months post end of treatment at study entry
• For those already at this timepoint, they are now eligible
• For those in treatment, or otherwise not yet at this timepoint, they are eligible once at they are = 6 months post end of treatment
• Prospective group patients must not have begun treatment
• Patients with diminished capacity will not be enrolled.
• Language: Patients must be able to communicate in English, French, or Spanish
• Sibling Cohort: One sibling, not affected by retinoblastoma will be enrolled, preference for the sibling closest in age to the RB patient.
• 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.

Related Conditions & MeSH terms

• Biological Sibling
• Cancer Survivor
• Intraocular Retinoblastoma
• Retinoblastoma
• Unilateral Retinoblastoma

Intervention

Procedure:
• Biospecimen collection
Collection of tissue and saliva samples

Other:
• Vision assessment
Undergo vision assessment
• Questionnaire administration
Complete questionnaires
• Quality of life assessment
Complete questionnaires
• Laboratory Biomarker Analysis
Correlative studies

Locations

Country	Name	City	State
Canada	The Hosptial for Sick Children	Toronto
United States	Lurie Children's Hospital	Chicago	Illinois
United States	University of Illinois, Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	MD Anderson Cancer Center	Houston	Texas
United States	Texas Childeren's Hospital	Houston	Texas
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin
United States	University of Minnesoa	Minneapolis	Minnesota
United States	Vanderbilt-Ingram Cancer Center	Nashville	Tennessee
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	Washington School of Medicine at St. Louis	Saint Louis	Missouri

Sponsors (2)

Lead Sponsor	Collaborator
Vanderbilt-Ingram Cancer Center	National Cancer Institute (NCI)

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of acute toxicity		Up to 1 year
Primary	Estimate malignant neoplasm (SMN) risk .Measured through medical record abstraction,		Up to 1 year
Primary	Assess visual outcomes measured via age appropriate visual acuity testing		Up to 1 year
Primary	Assess psycho-social outcomes utilizing questionnaires: BRIEF		Up to 2 years
Primary	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
Primary	Assess quality of life utilizing questionnaires: BRIEF		Up to 2 years
Primary	Assess quality of life utilizing questionnaires: CBCL		Up to 2 years
Primary	Assess quality of life utilizing questionnaires: Youth Self-Report		Up to 2 years
Primary	Assess quality of life utilizing questionnaires: Pediatric Quality of Life		Up to 2 years
Primary	Assess visual outcomes measured via parent report		Up to 1 year
Primary	Assess visual outcomes measured via vision questionnaires		Up to 1 year
Primary	Assess psycho-social outcomes utilizing questionnaires: BRIEF-P,		Up to 2 years
Primary	Assess psycho-social outcomes utilizing questionnaires: CBCL		Up to 2 years
Primary	Assess psycho-social outcomes utilizing questionnaires: Youth Self-Report		Up to 2 years
Primary	Assess psycho-social outcomes utilizing questionnaires: Pediatric Quality of Life		Up to 2 years