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

• ClinicalTrials.gov Identifier: NCT02520713
• Recruitment Status: Recruiting
• First Posted: August 13, 2015
• Last Update Posted: May 3, 2022
• Sponsor: Dana-Farber Cancer Institute
• Information provided by (Responsible Party): Katherine A. Janeway, Dana-Farber Cancer Institute

Tracking Information

• First Submitted Date: June 18, 2015
• First Posted Date: August 13, 2015
• Last Update Posted Date: May 3, 2022
• Study Start Date: October 2015
• Estimated Primary Completion Date: April 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 23, 2018)

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.

• Original Primary Outcome Measures (submitted: August 7, 2015): Overall Survival [ Time Frame: 18 months ]

Current Secondary Outcome Measures (submitted: August 7, 2015)

• Identification of the patient, clinical, and medication access factors associated with a) having an iCat recommendation and b) with receipt of matched targeted therapy. [ Time Frame: 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. [ Time Frame: 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. [ Time Frame: 2 Years ]
• 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. [ Time Frame: 2 Years ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study
• 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
• 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.

• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided

Biospecimen

Retention:   Samples With DNA

Description:

Tumor and Germline

• Sampling Method: Non-Probability Sample
• Study Population: High-risk, relapsed and refractory solid tumors
• Condition: 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.

Study Groups/Cohorts

Genetic testing and GAIN report

All enrolled patients will submit specimens for sequencing and analysis.

Intervention: Genetic: Genetic testing and GAIN report

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: August 7, 2015): 825
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: October 2025
• Estimated Primary Completion Date: April 2023 (Final data collection date for primary outcome measure)

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
• 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 30 Years (Child, Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Katherine Janeway, MD; 617-632-4994; KJANEWAY@PARTNERS.ORG

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02520713
• Other Study ID Numbers: 15-169
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No
• Plan Description: There is not plan to share IPD from this study at this time.

• Current Responsible Party: Katherine A. Janeway, Dana-Farber Cancer Institute
• Original Responsible Party: Katherine Janeway, MD, Dana-Farber Cancer Institute, Principal Investigator
• Current Study Sponsor: Dana-Farber Cancer Institute
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Katherine Janeway, MD; Dana-Farber Cancer Institute

• PRS Account: Dana-Farber Cancer Institute
• Verification Date: May 2022