Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood

• Conditions: Pediatric Cancer

• Registration Number: NCT02242552
• Lead Sponsor: St. Justine's Hospital

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-06
• Status Verified: 2014-08
• Study First Submitted: 2014-08-15
• Study First Submitted QC: 2014-09-15
• Last Update Submitted: 2014-09-15
• Study First Posted: 2014-09-17
• Last Update Posted: 2014-09-17
• Primary Completion: 2016-06
• Study Completion: 2017-06

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 30

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers.	24 months	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.

Secondary Outcome Measures

Name	Time	Method
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
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
Number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug.	24 months

Trial Locations

Locations (1)

CHU Sainte-Justine; 🇨🇦 Montréal, Quebec, Canada