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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02520713
Recruitment Status : Recruiting
First Posted : August 13, 2015
Last Update Posted : May 11, 2021
Information provided by (Responsible Party):
Katherine A. Janeway, Dana-Farber Cancer Institute

Tracking Information
First Submitted Date  ICMJE June 18, 2015
First Posted Date  ICMJE August 13, 2015
Last Update Posted Date May 11, 2021
Study Start Date  ICMJE October 2015
Estimated Primary Completion Date January 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (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  ICMJE
 (submitted: August 7, 2015)
Overall Survival [ Time Frame: 18 months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Condition  ICMJE Pediatric Solid Tumor
Intervention  ICMJE 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 Arms  ICMJE 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

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 7, 2015)
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE July 2024
Estimated Primary Completion Date January 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 30 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Katherine Janeway, MD 617-632-4994 KJANEWAY@PARTNERS.ORG
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT02520713
Other Study ID Numbers  ICMJE 15-169
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Plan Description: There is not plan to share IPD from this study at this time.
Responsible Party Katherine A. Janeway, Dana-Farber Cancer Institute
Study Sponsor  ICMJE Dana-Farber Cancer Institute
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Katherine Janeway, MD Dana-Farber Cancer Institute
PRS Account Dana-Farber Cancer Institute
Verification Date May 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP