Recruiting

Observational StudyStudying Health Outcomes After Treatment in Patients With Retinoblastoma

Sponsor:

Vanderbilt-Ingram Cancer Center

Code:

NCT03932786

Conditions

Retinoblastoma

Cancer Survivor

Biological Sibling

Intraocular Retinoblastoma

Unilateral Retinoblastoma

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Accepted

Interventions

Biospecimen collection

Vision assessment

Questionnaire administration

Quality of life assessment

Laboratory Biomarker Analysis

Study Details

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

Conditions

Retinoblastoma

Cancer Survivor

Biological Sibling

Intraocular Retinoblastoma

Unilateral Retinoblastoma

Study ID

NCT03932786

Start date

Jan 24, 2019

Status verified date

Sep, 2025

Completion date

Jan, 2031

Anticipated

Primary completion date

Jan, 2030

Anticipated

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Accepted

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 Design

Enrollment

900 participants

Anticipated

Interventions and Outcome Measures

Arms

Retrospective(biospecimens, vision assessment, questionnaires)

Prospective (biospecimens, vision assessment, questionnaires)

Interventions

Biospecimen collection

Collection of tissue and saliva samples

Vision assessment

Undergo vision assessment

Questionnaire administration

Complete questionnaires

Quality of life assessment

Complete questionnaires

Laboratory Biomarker Analysis

Correlative studies

Primary outcome measure

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 ]

Central Contacts and Locations

Central contacts

Vanderbilt-Ingram Service for Timely Access

800-811-8480 cip@vanderbilt.edu

Locations

Lurie Children's Hospital

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Monica Newmark

mnewmark@luriechildrens.org

Principal Investigator:

Joanna Weinstein, MD

University of Illinois, Chicago

Recruiting

Chicago, Illinois, United States, 60612

Contacts

Kristen kitsch

kitsc@uic.edu

Principal Investigator:

Mary Lou Schmidt, MD

University of Minnesoa

Recruiting

Minneapolis, Minnesota, United States, 55455

Contacts

Kendra Pallin

palli007@umn.edu

Principal Investigator:

Joseph Neglia, MD

Washington School of Medicine at St. Louis

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Kara Sauerburger

sauerburgerk@wustl.edu

Principal Investigator:

Robert Hayashi, MD

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Amanda Pfeiffer

amanda.pfeiffer@cchmc.org

Principal Investigator:

Rajaram Nagarajan, MD

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Stephen Markham

markhams@chop.edu

Principal Investigator:

Amish Shah, MD, PhD

Vanderbilt-Ingram Cancer Center

Recruiting

Nashville, Tennessee, United States, 37232

Contacts

Vanderbilt-Ingram Service for Timely Access

800-811-8480

MD Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Theresa Honey

TAHoney@mdanderson.org

Principal Investigator:

Anna Herzog, MD

Texas Childeren's Hospital

Recruiting

Houston, Texas, United States, 77030

Contacts

Najeeba Ali

nmali@texaschildrens.org

Principal Investigator:

Murali Chintagumpala, MD

Children's Hospital of Wisconsin

Recruiting

Milwaukee, Wisconsin, United States, 53226

Contacts

Veronica Seher

vseher@mcw.edu

Principal Investigator:

Cindy Schwartz, MD

The Hosptial for Sick Children

Recruiting

Toronto, Canada

Contacts

Roxanna Noronha

roxanna.noronha@sickkids.ca

Principal Investigator:

Helen Dimaras, MD

More Information

Sponsor

Vanderbilt-Ingram Cancer Center

Last update posted

Sep 19, 2025

Last verified

Sep, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Vanderbilt-Ingram Cancer Center on 2025-09-19.