Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood

Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood(TRICEPS Study)

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.

Study Overview

• Status: Unknown
• Conditions: Pediatric Cancer

• Study Type: Observational
• Enrollment (Anticipated): 30

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Montréal, Quebec, Canada, H3T1C5
      • Recruiting
      • CHU Sainte-Justine
      • Contact: Dominique Lafrenière, BScN, DESS; Phone Number: 514-345-4969; Email: dominique.lafreniere@recherche-ste-justine.qc.ca
      • Contact: Mary-Ellen French, RN; Phone Number: 514-345-4969; Email: mary-ellen.french@recherche-ste-justine.qc.ca

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 month to 21 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Children and adolescents with Relapse or refractory leukemia and solid tumor

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Only
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers.	The study team will evaluate the timeline between decision of biopsy, the actual biopsy, availability of results of the whole-genome analysis, interpretation of results and divulgation of results to patient and family.	24 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of children with cancer who are suitable candidates for targeted therapy at our institution each year.	24 months
Number and type of driver mutation(s) found in our population of recurrent or refractory cancers.	24 months
Number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug.	24 months
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	24 months

Collaborators and Investigators

• Sponsor: St. Justine's Hospital
• Investigators: Principal Investigator: Monia Marzouki, MD, St. Justine's Hospital

Study record dates

Study Major Dates

• Study Start: June 1, 2014
• Primary Completion (Anticipated): June 1, 2016
• Study Completion (Anticipated): June 1, 2017

Study Registration Dates

• First Submitted: August 15, 2014
• First Submitted That Met QC Criteria: September 15, 2014
• First Posted (Estimate): September 17, 2014

Study Record Updates

• Last Update Posted (Estimate): September 17, 2014
• Last Update Submitted That Met QC Criteria: September 15, 2014
• Last Verified: August 1, 2014

More Information

Terms related to this study

• Other Study ID Numbers: Triceps