Clinical trial NCT03932786: Studying Health Outcomes After Treatment in Patients With Retinoblastoma
Official title: Research Into Visual Endpoints and RB Health Outcomes After Treatment (RIVERBOAT)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Retinoblastoma
Cancer Survivor
Biological Sibling
Intraocular Retinoblastoma
Unilateral Retinoblastoma
Lead sponsor: Vanderbilt-Ingram Cancer Center
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
First posted: 2019-05-01
Enrollment: 900
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: OTHER
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

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

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.

Eligibility criteria: * 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.

Primary outcomes:
measure: Incidence of acute toxicity
timeFrame: Up to 1 year
measure: Estimate malignant neoplasm (SMN) risk .Measured through medical record abstraction,
timeFrame: Up to 1 year
measure: Assess visual outcomes measured via age appropriate visual acuity testing
timeFrame: Up to 1 year
measure: Assess psycho-social outcomes utilizing questionnaires: BRIEF
timeFrame: Up to 2 years
measure: 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.
timeFrame: Up to 1 year
measure: Assess quality of life utilizing questionnaires: BRIEF
timeFrame: Up to 2 years
measure: Assess quality of life utilizing questionnaires: CBCL
timeFrame: Up to 2 years
measure: Assess quality of life utilizing questionnaires: Youth Self-Report
timeFrame: Up to 2 years
measure: Assess quality of life utilizing questionnaires: Pediatric Quality of Life
timeFrame: Up to 2 years
measure: Assess visual outcomes measured via parent report
timeFrame: Up to 1 year
measure: Assess visual outcomes measured via vision questionnaires
timeFrame: Up to 1 year
measure: Assess psycho-social outcomes utilizing questionnaires: BRIEF-P,
timeFrame: Up to 2 years
measure: Assess psycho-social outcomes utilizing questionnaires: CBCL
timeFrame: Up to 2 years
measure: Assess psycho-social outcomes utilizing questionnaires: Youth Self-Report
timeFrame: Up to 2 years
measure: Assess psycho-social outcomes utilizing questionnaires: Pediatric Quality of Life
timeFrame: Up to 2 years
Secondary outcomes:
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Lurie Children's Hospital
state: Illinois
city: Chicago
country: United States
facility: University of Illinois, Chicago
state: Illinois
city: Minneapolis
country: United States
facility: University of Minnesoa
state: Minnesota
city: Saint Louis
country: United States
facility: Washington School of Medicine at St. Louis
state: Missouri
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt-Ingram Cancer Center
state: Tennessee
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Houston
country: United States
facility: Texas Childeren's Hospital
state: Texas
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Toronto
country: Canada
facility: The Hosptial for Sick Children
Study officials:
affiliation: Vanderbilt Medical Center
name: Debra Friedman, MD
role: PRINCIPAL_INVESTIGATOR
References: