for people ages up to 30 years (full criteria)
at San Francisco, California and other locations
study started
completion around
Principal Investigator
by Amit Sabnis
Headshot of Amit Sabnis
Amit Sabnis



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.

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


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.


Pediatric Solid Tumor, Genetic testing and GAIN report


You can join if…

Open to people ages up to 30 years

  • 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 .

You CAN'T join if...

  • 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.


  • UCSF
    San Francisco California 94158 United States
  • University of Utah Childrens Medical Center
    Salt Lake City Utah 84113 United States

Lead Scientist at UCSF

  • Amit Sabnis
    I am a pediatric oncologist and physician-scientist with a focus on examining new therapeutic strategies for sarcomas.


in progress, not accepting new patients
Start Date
Completion Date
Dana-Farber Cancer Institute
Study Type
Expecting 825 study participants
Last Updated