Clinical trial NCT02242552: Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood
Official title: Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood(TRICEPS Study)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Pediatric Cancer
Lead sponsor: St. Justine's Hospital
Sponsor class: OTHER
First posted: 2014-09-17
Enrollment: 30
Sex: ALL
Minimum age (years): 1
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children and adolescents with Relapse or refractory leukemia and solid tumor

Brief summary: A new research paradigm that involves sequencing tumor DNA/RNA to identify driver mutations, select among the Health Canada approved drugs (for adult cancers) known to block certain oncogenic pathways, and recommend these drugs to the treating physician, without taking into account the tumor histology.

In this paradigm, the treatment is targeted to the actionable mutation(s) i.e. those driving oncogenesis. It is also personalized to the molecular signature of the patient's tumor, irrespective of its histopathological subtype. The experience of the investigators team in genomics, including next generation sequencing and bioinformatic analysis combined with the clinical expertise, bring at last this approach within our technical capacities. In parallel, the number of Health Canada-approved drugs (which have been tested in a pediatric setting) designed to interfere with oncogenesis pathways is increasing exponentially.


Eligibility criteria: Inclusion Criteria:

At the time of enrollment:

* 21 year-old or less
* Poor prognosis biopsy-proven cancer of any type :
* Cancer (at initial diagnosis) known to be refractory to treatment
* Or cancer refractory to treatment
* Or relapsed cancer
* Written informed consent by patient, parents, or the legal guardians

Exclusion Criteria:

* Estimated life expectancy of less than 3 months.

Primary outcomes:
measure: Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers.
timeFrame: 24 months
Secondary outcomes:
measure: Number of children with cancer who are suitable candidates for targeted therapy at our institution each year.
timeFrame: 24 months
measure: Number and type of driver mutation(s) found in our population of recurrent or refractory cancers.
timeFrame: 24 months
measure: Number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug.
timeFrame: 24 months
measure: Feasibility of performing whole genome sequencing and data analysis, identifying a drug based on the genomic data and offering this information to the medical team, the patient and the family within 10-week time frame from diagnosis
timeFrame: 24 months
Other outcomes:
Locations:
city: Montréal
country: Canada
facility: CHU Sainte-Justine
state: Quebec
Study officials:
affiliation: St. Justine's Hospital
name: Monia Marzouki, MD
role: PRINCIPAL_INVESTIGATOR
References: