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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Pediatric Solid Tumor
• Intervention / Treatment: 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.

• Study Type: Observational
• Enrollment (Estimated): 825

Contacts and Locations

Study Locations

• United States
  • California
    • San Francisco, California, United States, 94158
      • University of California San Francisco
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital Colorado
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Children's National Medical Center
  • Illinois
    • Chicago, Illinois, United States, 60637
      • University of Chicago Comer Children's Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana-Farber Cancer Institute
    • Boston, Massachusetts, United States, 02215
      • Boston Children's Hospital
  • New York
    • New York, New York, United States, 10032
      • Columbia University Medical Center
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Texas
    • Dallas, Texas, United States, 75390
      • University of Texas Southwestern Medical Center at Dallas
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • University of Utah Childrens Medical Center
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 30 years (Child, Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

High-risk, relapsed and refractory solid tumors

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival	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.	18 months

Secondary Outcome Measures

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

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2015
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): October 1, 2026

Study Registration Dates

• First Submitted: June 18, 2015
• First Submitted That Met QC Criteria: August 7, 2015
• First Posted (Estimated): August 13, 2015

Study Record Updates

• Last Update Posted (Actual): April 28, 2026
• Last Update Submitted That Met QC Criteria: April 23, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Pediatric Solid Tumor
• Additional Relevant MeSH Terms: Investigative Techniques; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Health Services; Health Care Facilities Workforce and Services; Preventive Health Services; Genetic Techniques; Genetic Services; Diagnostic Services; Genetic Testing
• Other Study ID Numbers: 15-169

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: There is not plan to share IPD from this study at this time.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No