Multicenter Cohort Study To Evaluate Outcomes After Receipt of Targeted Therapy Matched to an Individualized Cancer Therapy (iCat) Recommendations in Children and Young Adults With Solid Tumors: The iCat2, GAIN Consortium Study
Overview
- Phase
- N/A
- Intervention
- Genetic testing and GAIN report
- Conditions
- Pediatric Solid Tumor
- Sponsor
- Dana-Farber Cancer Institute
- Enrollment
- 825
- Locations
- 24
- Primary Endpoint
- Overall Survival
- Status
- Active, Not Recruiting
- Last Updated
- 4 days ago
Overview
Brief Summary
This research study is evaluating the use of specialized testing of solid tumors including sequencing. The process of performing these specialized tests is called tumor profiling. The tumor profiling may result in identifying changes in genes of the tumor that indicate that a particular therapy may have activity. This is called an individualized cancer therapy (iCat) recommendation. The results of the tumor profiling and, if applicable, the iCat recommendation will be returned.
Detailed Description
Patients with extra-cranial (not in the brain) solid tumors that are either difficult to diagnose or more difficult to treat are eligible to participate in this study. All enrolled patients will have targeted sequencing of tumor performed. Sequencing results will be reviewed for clinically significant findings including determination of whether any mutations exist that suggest potential for activity of a targeted therapy (iCat recommendation). Results will be returned to the patient's oncologist and follow-up data will be collected. In this prospective multi-center cohort study, the primary objective is to describe the outcomes of pediatric patients with advanced solid tumors according to whether or not they received of targeted therapy matched to an iCat recommendation. The primary clinical outcome of interest is the endpoint of overall survival (OS), with progression-free survival and response rate (RR) as key secondary clinical outcome measures. To address this hypothesis, 825 patients will enroll from an anticipated 11 participating institutions over 3 years.
Investigators
Katherine A. Janeway, MD
Principal Investigator
Dana-Farber Cancer Institute
Eligibility Criteria
Inclusion Criteria
- •Age -- Age ≤ 30 years at time of initial qualifying solid tumor diagnosis
- •Diagnosis -- Histologic diagnosis of solid malignancy (excluding brain tumors and lymphoma) that meets at least one of the following criteria:
- •Refractory, defined as tumor progression after initiation of standard first line therapy without having achieved a prior partial or complete remission OR Biopsy proven residual disease at the completion of planned standard initial front-line therapy.
- •Recurrent, defined as tumor progression after achieving a prior partial or complete remission
- •Newly diagnosed high risk disease, defined as having an expected event free survival of \< 50% at 2 years.
- •Lacks definitive diagnosis or classical genomic findings after histologic review and standard molecular testing (rare tumor group).
- •Examples include (eligibility not limited to these examples):
- •Histology typically associated with a fusion in which fusion is not detected.
- •Ewing-like sarcoma
- •Undifferentiated sarcoma
Exclusion Criteria
- •No Therapy Planned
- •\-- Patients who have declined further anticancer therapy will be excluded.
- •Performance Status
- •\-- Patients with Lansky (age \< 16 years) or Karnofsky (age ≥16 years) score \< 50 will be excluded.
- •Life Expectancy -- Patients with anticipated life expectancy \< 3 months will be excluded.
Arms & Interventions
Genetic testing and GAIN report
All enrolled patients will submit specimens for sequencing and analysis.
Intervention: Genetic testing and GAIN report
Outcomes
Primary Outcomes
Overall Survival
Time Frame: 18 months
In pediatric patients with recurrent or refractory solid tumors who undergo successful molecular profiling, a) to describe the overall survival of patients by treatment group (iCatM, iCatUM and UM); b) to identify demographic, clinical, and genomic factors associated with overall survival from the time of relapse/progression (OSr); and c)to store tumor material, derived cell lines / xenografts, and blood samples for general sample banking and potential future research.
Secondary Outcomes
- Determination of whether participation in a genomics study provides psychological well-being and whether that is associated with level of genomic comprehension and with receipt of an iCat recommendation.(2 Years)
- Determination of factors associated with response and progression-free survival time by treatment group for patients with recurrent/refractory disease and measurable/evaluable disease.(2 Years)
- Identification of the patient, clinical, and medication access factors associated with a) having an iCat recommendation and b) with receipt of matched targeted therapy.(2 Years)
- Description of the frequency and range of molecular alterations in pediatric solid tumors at diagnosis and at relapse including a comparison of potentially targetable variants in paired tumor samples obtained from relapse and at initial diagnosis.(2 Years)