RetinoblastomaRIVERBOATNCT03932786
Studying Health Outcomes After Treatment in Patients With Retinoblastoma
An observational study of Retinoblastoma and Cancer Survivor, run by Vanderbilt-Ingram Cancer Center at 11 sites in 2 countries.
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… Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Retinoblastoma, Cancer Survivor, Biological Sibling
- What is being tested?
- Biospecimen collection, Vision assessment and Questionnaire administration
- Type of treatment
- Surgery or procedure
- How many people
- 900 participants
- Where
- 11 sitesin 2 countries
- Study dates
- 24 Jan 2019to Jan 2031
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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
- 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.
Trial locations and contacts
11 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
11 sites in 2 countries
United States10 sites, 10 recruiting
- Recruiting
Lurie Children's Hospital
Chicago, Illinois, 60611
Monica Newmark, mnewmark@luriechildrens.org
Joanna Weinstein, MD
- Recruiting
University of Illinois, Chicago
Chicago, Illinois, 60612
Kristen kitsch, kitsc@uic.edu
Mary Lou Schmidt, MD
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Amanda Pfeiffer, amanda.pfeiffer@cchmc.org
Rajaram Nagarajan, MD
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Theresa Honey, TAHoney@mdanderson.org
Anna Herzog, MD
- Recruiting
Texas Childeren's Hospital
Houston, Texas, 77030
Najeeba Ali, nmali@texaschildrens.org
Murali Chintagumpala, MD
- Recruiting
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Veronica Seher, vseher@mcw.edu
Cindy Schwartz, MD
- Recruiting
University of Minnesoa
Minneapolis, Minnesota, 55455
Kendra Pallin, palli007@umn.edu
Joseph Neglia, MD
- Recruiting
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, 37232
Vanderbilt-Ingram Service for Timely Access, 800-811-8480
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Stephen Markham, markhams@chop.edu
Amish Shah, MD, PhD
- Recruiting
Washington School of Medicine at St. Louis
St Louis, Missouri, 63110
Kara Sauerburger, sauerburgerk@wustl.edu
Robert Hayashi, MD
Canada1 site, 1 recruiting
- Recruiting
The Hosptial for Sick Children
Toronto
Roxanna Noronha, roxanna.noronha@sickkids.ca
Helen Dimaras, MD
About this study
Official title: 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
Read the detailed description
PRIMARY OBJECTIVES:
- 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.
- Conditions
- Retinoblastoma, Cancer Survivor, Biological Sibling, Intraocular Retinoblastoma, Unilateral Retinoblastoma
- Being tested
- Biospecimen collection, Vision assessment, Questionnaire administration, Quality of life assessment, Laboratory Biomarker Analysis
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen collectionProcedure or surgery
Collection of tissue and saliva samples
Vision assessmentOther
Undergo vision assessment
Questionnaire administrationOther
Complete questionnaires
Quality of life assessmentOther
Complete questionnaires
Laboratory Biomarker AnalysisOther
Correlative studies
| Study group | Role | What they receive |
|---|---|---|
| Retrospective(biospecimens, vision assessment, questionnaires) | Not described Receives: Procedure: Biospecimen collection; Other: Vision assessment; Other: Questionnaire administration; Other: Quality of life assessment; Other: Laboratory Biomarker Analysis | |
| Prospective (biospecimens, vision assessment, questionnaires) | Not described Receives: Procedure: Biospecimen collection; Other: Vision assessment; Other: Questionnaire administration; Other: Quality of life assessment; Other: Laboratory Biomarker Analysis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of acute toxicity
Measured: Up to 1 year
Estimate malignant neoplasm (SMN) risk .Measured through medical record abstraction,
Measured: Up to 1 year
Assess visual outcomes measured via age appropriate visual acuity testing
Measured: Up to 1 year
Assess psycho-social outcomes utilizing questionnaires: BRIEF
Measured: 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.
Measured: Up to 1 year
Assess quality of life utilizing questionnaires: BRIEF
Measured: Up to 2 years
Assess quality of life utilizing questionnaires: CBCL
Measured: Up to 2 years
Assess quality of life utilizing questionnaires: Youth Self-Report
Measured: Up to 2 years
Assess quality of life utilizing questionnaires: Pediatric Quality of Life
Measured: Up to 2 years
Assess visual outcomes measured via parent report
Measured: Up to 1 year
Assess visual outcomes measured via vision questionnaires
Measured: Up to 1 year
Assess psycho-social outcomes utilizing questionnaires: BRIEF-P,
Measured: Up to 2 years
Assess psycho-social outcomes utilizing questionnaires: CBCL
Measured: Up to 2 years
Assess psycho-social outcomes utilizing questionnaires: Youth Self-Report
Measured: Up to 2 years
Assess psycho-social outcomes utilizing questionnaires: Pediatric Quality of Life
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2019
- Study start24 Jan 2019
- Primary completionJan 2030 (estimated)
- Study completionJan 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RetinoblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Other
- Enrolment
- 900 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Vanderbilt-Ingram Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Principal investigator, Debra Friedman, Vanderbilt-Ingram Cancer Center
- Investigators
- Debra Friedman, MD, Principal investigator, Vanderbilt Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03932786
- Sponsor's protocol ID
- VICC PED 1878
- Other IDs
- R01CA225005, NCI-2019-00635
- Acronym
- RIVERBOAT
- Status last verified
- Sep 2025
- First submitted
- 15 Mar 2019
- Last updated
- 19 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Research Into Visual Endpoints and RB Health Outcomes After Treatment (RIVERBOAT)
- ClinicalTrials.gov ID
- NCT03932786
- Acronym / sponsor ID
- RIVERBOAT / VICC PED 1878
- Conditions
- Retinoblastoma, Cancer Survivor, Biological Sibling, Intraocular Retinoblastoma, Unilateral Retinoblastoma
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 900 participants (estimated)
- Lead sponsor
- Vanderbilt-Ingram Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 24 Jan 2019 (actual)
- Primary completion
- Jan 2030 (estimated)
- Study completion
- Jan 2031 (estimated)
- First posted
- 1 May 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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 design
- Observational model
- Case-control
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Retrospective(biospecimens, vision assessment, questionnaires) | – | Procedure: Biospecimen collection, Other: Vision assessment, Other: Questionnaire administration, Other: Quality of life assessment, Other: Laboratory Biomarker Analysis |
| Prospective (biospecimens, vision assessment, questionnaires) | – | Procedure: Biospecimen collection, Other: Vision assessment, Other: Questionnaire administration, Other: Quality of life assessment, Other: Laboratory Biomarker Analysis |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biospecimen collectionCollection of tissue and saliva samples | Procedure or surgery | Prospective (biospecimens, vision assessment, questionnaires), Retrospective(biospecimens, vision assessment, questionnaires) |
| Vision assessmentUndergo vision assessment | Other | Prospective (biospecimens, vision assessment, questionnaires), Retrospective(biospecimens, vision assessment, questionnaires) |
| Questionnaire administrationComplete questionnaires | Other | Prospective (biospecimens, vision assessment, questionnaires), Retrospective(biospecimens, vision assessment, questionnaires) |
| Quality of life assessmentComplete questionnaires | Other | Prospective (biospecimens, vision assessment, questionnaires), Retrospective(biospecimens, vision assessment, questionnaires) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Prospective (biospecimens, vision assessment, questionnaires), Retrospective(biospecimens, vision assessment, questionnaires) |
Primary outcome
Incidence of acute toxicity
Time frame: Up to 1 year
Estimate malignant neoplasm (SMN) risk .Measured through medical record abstraction,
Time frame: Up to 1 year
Assess visual outcomes measured via age appropriate visual acuity testing
Time frame: Up to 1 year
Assess psycho-social outcomes utilizing questionnaires: BRIEF
Time frame: 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
Assess quality of life utilizing questionnaires: BRIEF
Time frame: Up to 2 years
Assess quality of life utilizing questionnaires: CBCL
Time frame: Up to 2 years
Assess quality of life utilizing questionnaires: Youth Self-Report
Time frame: Up to 2 years
Assess quality of life utilizing questionnaires: Pediatric Quality of Life
Time frame: Up to 2 years
Assess visual outcomes measured via parent report
Time frame: Up to 1 year
Assess visual outcomes measured via vision questionnaires
Time frame: Up to 1 year
Assess psycho-social outcomes utilizing questionnaires: BRIEF-P,
Time frame: Up to 2 years
Assess psycho-social outcomes utilizing questionnaires: CBCL
Time frame: Up to 2 years
Assess psycho-social outcomes utilizing questionnaires: Youth Self-Report
Time frame: Up to 2 years
Assess psycho-social outcomes utilizing questionnaires: Pediatric Quality of Life
Time frame: Up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2025.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Debra Friedman, MD principal investigator
Vanderbilt Medical Center
11 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Retinoblastoma and Cancer Survivor, run by Vanderbilt-Ingram Cancer Center at 11 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Vanderbilt-Ingram Service for Timely Access (800-811-8480, cip@vanderbilt.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of acute toxicity (time frame: Up to 1 year). The study lists 15 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2025.