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 25, 2023
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Condition or disease | Intervention/treatment |
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Pediatric Solid Tumor | Genetic: Genetic testing and GAIN report |
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 |
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 |

Group/Cohort | Intervention/treatment |
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Genetic testing and GAIN report
All enrolled patients will submit specimens for sequencing and analysis.
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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. |
- 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.
- 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 ]
Biospecimen Retention: Samples With DNA

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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 |
Inclusion Criteria:
- Age -- Age ≤ 30 years at time of initial qualifying solid tumor diagnosis
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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
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Specimen Samples
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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 .
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Exclusion Criteria:
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No Therapy Planned
-- Patients who have declined further anticancer therapy will be excluded.
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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.

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 ClinicalTrials.gov identifier (NCT number): NCT02520713
Contact: Katherine Janeway, MD | 617-632-4994 | KJANEWAY@PARTNERS.ORG |
United States, California | |
University of California San Francisco | Recruiting |
San Francisco, California, United States, 94158 | |
Contact: Amit Sabnis, MD 415-476-3831 Amit.Sabnis@ucsf.edu | |
United States, Colorado | |
Children's Hospital Colorado | Recruiting |
Aurora, Colorado, United States, 80045 | |
Contact: Margaret Macy, MD 720-777-6772 margaret.macy@childrenscolorado.org | |
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 kjaneway@partners.org | |
Principal Investigator: Katherine Janeway, MD | |
Dana-Farber Cancer Institute | Recruiting |
Boston, Massachusetts, United States, 02215 | |
Contact: Katherine Janeway, MD 617-632-4994 kjaneway@partners.org | |
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 Susan.Colace@nationwidechildrens.org | |
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 Laura.Klesse@UTSouthwestern.edu | |
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 navin.pinto@seattlechildrens.org |
Principal Investigator: | Katherine Janeway, MD | Dana-Farber Cancer Institute |
Responsible Party: | Katherine A. Janeway, MD, Principal Investigator, Dana-Farber Cancer Institute |
ClinicalTrials.gov 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. |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Pediatric Solid Tumor |