Studying Health Outcomes After Treatment in Patients With Retinoblastoma (RIVERBOAT)

September 15, 2025 updated by: Debra Friedman, Vanderbilt-Ingram Cancer Center

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Estimated)

900

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Vanderbilt-Ingram Service for Timely Access
  • Phone Number: 800-811-8480
  • Email: cip@vanderbilt.edu

Study Locations

  • Canada
      • Toronto, Canada
        • Recruiting
        • The Hosptial for Sick Children
        • Contact:
        • Principal Investigator:
          • Helen Dimaras, MD
  • United States
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Lurie Children's Hospital
        • Contact:
        • Principal Investigator:
          • Joanna Weinstein, MD
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • University of Illinois, Chicago
        • Contact:
        • Principal Investigator:
          • Mary Lou Schmidt, MD
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesoa
        • Contact:
        • Principal Investigator:
          • Joseph Neglia, MD
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • Washington School of Medicine at St. Louis
        • Contact:
        • Principal Investigator:
          • Robert Hayashi, MD
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital Medical Center
        • Contact:
        • Principal Investigator:
          • Rajaram Nagarajan, MD
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Contact:
        • Principal Investigator:
          • Amish Shah, MD, PhD
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt-Ingram Cancer Center
        • Contact:
          • Vanderbilt-Ingram Service for Timely Access
          • Phone Number: 800-811-8480
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • MD Anderson Cancer Center
        • Contact:
        • Principal Investigator:
          • Anna Herzog, MD
      • Houston, Texas, United States, 77030
        • Recruiting
        • Texas Childeren's Hospital
        • Contact:
        • Principal Investigator:
          • Murali Chintagumpala, MD
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Children's Hospital of Wisconsin
        • Contact:
        • Principal Investigator:
          • Cindy Schwartz, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

Yes

Sampling Method

Non-Probability Sample

Study Population

Retrospective patients (those treated between 2008 and 2018) identified by each site at the start of the study period and prospective patients (those treated between 2018 and 2023) identified at the time of diagnosis by each site during the study period with unilateral or bilateral intraocular retinoblastoma

Description

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Retrospective(biospecimens, vision assessment, questionnaires)
Other: Laboratory Biomarker Analysis
Correlative studies
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
Prospective (biospecimens, vision assessment, questionnaires)
Other: Laboratory Biomarker Analysis
Correlative studies
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

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vanderbilt-Ingram Cancer Center

Collaborators

National Cancer Institute (NCI)

Investigators

  • Principal Investigator: Debra Friedman, MD, Vanderbilt Medical Center

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 24, 2019

Primary Completion (Estimated)

January 1, 2030

Study Completion (Estimated)

January 1, 2031

Study Registration Dates

First Submitted

March 15, 2019

First Submitted That Met QC Criteria

April 27, 2019

First Posted (Actual)

May 1, 2019

Study Record Updates

Last Update Posted (Estimated)

September 19, 2025

Last Update Submitted That Met QC Criteria

September 15, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VICC PED 1878
  • NCI-2019-00635 (Registry Identifier: NCI, Clinical Trials Reporting Program)
  • R01CA225005 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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