The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study

Pediatric Solid Tumor Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02520713
• Other study ID #: 15-169
• Status: Active, not recruiting
• Start date: October 2015
• Est. completion date: October 2025

Study information

• Verified date: January 2024
• Source: Dana-Farber Cancer Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 825
• Est. completion date: October 2025
• Est. primary completion date: January 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 30 Years

Eligibility

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
• Inflammatory myofibroblastic tumor without ALK fusion
• Infantile fibrosarcoma without NTRK fusion
• Specimen Samples
• Sufficient tumor specimen available to meet the minimum requirements for profiling from diagnosis or progression / recurrence --- OR
• Surgery / biopsy planned as part of clinical care that is anticipated to yield sufficient material to meet the minimum requirements for profiling; OR
• Patient has already had molecular profiling and patient has not yet started matched targeted therapy based on the report .

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.

Related Conditions & MeSH terms

• Pediatric Solid Tumor

Intervention

Genetic:
• Genetic testing and GAIN report
All patients enrolled will submit specimens for genetic analysis. If successful the report will be reviewed to look for possible recommendations and a GAIN report will be generated regardless of possible recommendations.

Locations

Country	Name	City	State
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Dana-Farber Cancer Institute	Boston	Massachusetts
United States	University of Chicago Comer Children's Hospital	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of Texas Southwestern Medical Center at Dallas	Dallas	Texas
United States	Columbia University Medical Center	New York	New York
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	University of Utah Childrens Medical Center	Salt Lake City	Utah
United States	University of California San Francisco	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Dana-Farber Cancer Institute

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall Survival	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.	18 months
Secondary	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
Secondary	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
Secondary	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
Secondary	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