Clinical trial NCT02520713: 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
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Pediatric Solid Tumor
Keywords: Pediatric Solid Tumor
Lead sponsor: Dana-Farber Cancer Institute
Sponsor class: OTHER
First posted: 2015-08-13
Enrollment: 825
Sex: ALL
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: High-risk, relapsed and refractory solid tumors

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.

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.

Primary outcomes:
measure: Overall Survival
timeFrame: 18 months
Secondary outcomes:
measure: Identification of the patient, clinical, and medication access factors associated with a) having an iCat recommendation and b) with receipt of matched targeted therapy.
timeFrame: 2 Years
measure: Determination of factors associated with response and progression-free survival time by treatment group for patients with recurrent/refractory disease and measurable/evaluable disease.
timeFrame: 2 Years
measure: 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.
timeFrame: 2 Years
measure: 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.
timeFrame: 2 Years
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Chicago
country: United States
facility: University of Chicago Comer Children's Hospital
state: Illinois
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center at Dallas
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Childrens Medical Center
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: Dana-Farber Cancer Institute
name: Katherine Janeway, MD
role: PRINCIPAL_INVESTIGATOR
References: