Studying Health Outcomes After Treatment in Patients With Retinoblastoma

Официальное название

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

Обзор

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

Подробное описание

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.

Вмешательства

• Процедура Biospecimen collection
  Collection of tissue and saliva samples
• Другое Vision assessment
  Undergo vision assessment
• Другое Questionnaire administration
  Complete questionnaires
• Другое Quality of life assessment
  Complete questionnaires
• Другое Laboratory Biomarker Analysis
  Correlative studies

Первичные конечные точки

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

Критерии участия

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Да

Дизайн исследования

Модель наблюдения

Случай-контроль

Центры проведения

США · 10 центров

• Lurie Children's Hospital — Chicago
• University of Illinois, Chicago — Chicago
• University of Minnesoa — Minneapolis
• Washington School of Medicine at St. Louis — St Louis
• Cincinnati Children's Hospital Medical Center — Cincinnati
• Children's Hospital of Philadelphia — Philadelphia
• Vanderbilt-Ingram Cancer Center — Nashville
• MD Anderson Cancer Center — Houston
• … и ещё 2 центра

Канада · 1 центр

• The Hosptial for Sick Children — Toronto

Идентификаторы

NCT: NCT03932786 · VICC PED 1878 · R01CA225005 · NCI-2019-00635

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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