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

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ClinicalTrials.gov Identifier: NCT02520713
Recruitment Status : Recruiting
First Posted : August 13, 2015
Last Update Posted : May 3, 2022
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 ]
Change History
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
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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: August 7, 2015)
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.
Sexes Eligible for Study: All
Ages up to 30 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contact: Katherine Janeway, MD 617-632-4994 KJANEWAY@PARTNERS.ORG
Listed Location Countries United States
Removed Location Countries  
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
Principal Investigator: Katherine Janeway, MD Dana-Farber Cancer Institute
PRS Account Dana-Farber Cancer Institute
Verification Date May 2022