Studying Health Outcomes After Treatment in Patients With Retinoblastoma

NCT03932786 | Recruiting DMC

• 3 other identifiers: VICC PED 1878R01CA225005NCI-2019-00635
• Study Type: observational
• Target: 900
• Locations: 2 countries
• Sites: 11

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

Outcome Measures

Primary Outcomes (15)

• Incidence of acute toxicity

  Up to 1 year

• Estimate malignant neoplasm (SMN) risk .Measured through medical record abstraction,

  Up to 1 year

• Assess visual outcomes measured via age appropriate visual acuity testing

  Up to 1 year

• Assess psycho-social outcomes utilizing questionnaires: BRIEF

  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.

  Up to 1 year

• Assess quality of life utilizing questionnaires: BRIEF

  Up to 2 years

• Assess quality of life utilizing questionnaires: CBCL

  Up to 2 years

• Assess quality of life utilizing questionnaires: Youth Self-Report

  Up to 2 years

• Assess quality of life utilizing questionnaires: Pediatric Quality of Life

  Up to 2 years

• Assess visual outcomes measured via parent report

  Up to 1 year

• Assess visual outcomes measured via vision questionnaires

  Up to 1 year

• Assess psycho-social outcomes utilizing questionnaires: BRIEF-P,

  Up to 2 years

• Assess psycho-social outcomes utilizing questionnaires: CBCL

  Up to 2 years

• Assess psycho-social outcomes utilizing questionnaires: Youth Self-Report

  Up to 2 years

• Assess psycho-social outcomes utilizing questionnaires: Pediatric Quality of Life

  Up to 2 years

Study Arms (2)

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

Interventions

Biospecimen collection PROCEDURE

Collection of tissue and saliva samples

Prospective (biospecimens, vision assessment, questionnaires); Retrospective(biospecimens, vision assessment, questionnaires)

Vision assessment OTHER

Undergo vision assessment

Prospective (biospecimens, vision assessment, questionnaires); Retrospective(biospecimens, vision assessment, questionnaires)

Questionnaire administration OTHER

Complete questionnaires

Prospective (biospecimens, vision assessment, questionnaires); Retrospective(biospecimens, vision assessment, questionnaires)

Quality of life assessment OTHER

Complete questionnaires

Prospective (biospecimens, vision assessment, questionnaires); Retrospective(biospecimens, vision assessment, questionnaires)

Laboratory Biomarker Analysis OTHER

Correlative studies

Prospective (biospecimens, vision assessment, questionnaires); Retrospective(biospecimens, vision assessment, questionnaires)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

* 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.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Vanderbilt-Ingram Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (11)

Lurie Children's Hospital

Chicago, Illinois, 60611, United States

RECRUITING

University of Illinois, Chicago

Chicago, Illinois, 60612, United States

RECRUITING

University of Minnesoa

Minneapolis, Minnesota, 55455, United States

RECRUITING

Washington School of Medicine at St. Louis

St Louis, Missouri, 63110, United States

RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Vanderbilt-Ingram Cancer Center

Nashville, Tennessee, 37232, United States

RECRUITING

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Texas Childeren's Hospital

Houston, Texas, 77030, United States

RECRUITING

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

RECRUITING

The Hosptial for Sick Children

Toronto, Canada

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Tissue, saliva

MeSH Terms

Conditions

Retinoblastoma

Condition Hierarchy (Ancestors)

Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Retinal Neoplasms; Eye Neoplasms; Neoplasms by Site; Eye Diseases, Hereditary; Eye Diseases; Retinal Diseases

Study Officials

• Debra Friedman, MD

  Vanderbilt Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Vanderbilt-Ingram Service for Timely Access

CONTACT

800-811-8480; cip@vanderbilt.edu

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Sponsor Investigator

Study Record Dates

• First Submitted: March 15, 2019
• First Posted: May 1, 2019
• Study Start: January 24, 2019
• Primary Completion (Estimated): January 1, 2030
• Study Completion (Estimated): January 1, 2031
• Last Updated: September 19, 2025

Record last verified: 2025-09

Locations

CA (1); US (10)