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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 25, 2023
Information provided by (Responsible Party):
Katherine A. Janeway, MD, Dana-Farber Cancer Institute

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.

Condition or disease Intervention/treatment
Pediatric Solid Tumor Genetic: Genetic testing and GAIN report

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.

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Study Type : Observational
Estimated Enrollment : 825 participants
Observational Model: Cohort
Time Perspective: Prospective
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
Study Start Date : October 2015
Estimated Primary Completion Date : September 2023
Estimated Study Completion Date : October 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Genetic Testing

Group/Cohort Intervention/treatment
Genetic testing and GAIN report
All enrolled patients will submit specimens for sequencing and analysis.
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.

Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. 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 ]

Biospecimen Retention:   Samples With DNA
Tumor and Germline

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
High-risk, relapsed and refractory solid tumors

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02520713

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

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United States, California
University of California San Francisco Recruiting
San Francisco, California, United States, 94158
Contact: Amit Sabnis, MD    415-476-3831   
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Margaret Macy, MD    720-777-6772   
United States, District of Columbia
Children's National Medical Center Recruiting
Washington, District of Columbia, United States, 20010
Contact: AeRang Kim, MD,PhD         
United States, Illinois
University of Chicago Comer Children's Hospital Recruiting
Chicago, Illinois, United States, 60637
Contact: Mark Applebaum, MD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02215
Contact: Katherine Janeway, MD    617-632-4994   
Principal Investigator: Katherine Janeway, MD         
Dana-Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02215
Contact: Katherine Janeway, MD    617-632-4994   
United States, New York
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
Contact: Darrell Yamashiro, MD, PhD         
United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Susan Colace, MD    614-722-2495   
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Rochelle Bagatell, MD         
United States, Texas
University of Texas Southwestern Medical Center at Dallas Recruiting
Dallas, Texas, United States, 75390
Contact: Laura Klesse, MD    214-456-6405   
United States, Utah
University of Utah Childrens Medical Center Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Luke Maese, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Navin Pinto, MD    206-987-2106   
Sponsors and Collaborators
Dana-Farber Cancer Institute
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Principal Investigator: Katherine Janeway, MD Dana-Farber Cancer Institute
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Responsible Party: Katherine A. Janeway, MD, Principal Investigator, Dana-Farber Cancer Institute Identifier: NCT02520713    
Other Study ID Numbers: 15-169
First Posted: August 13, 2015    Key Record Dates
Last Update Posted: May 25, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: There is not plan to share IPD from this study at this time.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Katherine A. Janeway, MD, Dana-Farber Cancer Institute:
Pediatric Solid Tumor