Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood

Sponsor

St. Justine's Hospital (Other)

Overall Status

Unknown status

CT.gov ID

NCT02242552

Collaborator

(none)

Enrollment

Location

Duration (Months)

0.8

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Pediatric Cancer

Study Design

Study Type:

Observational

Anticipated Enrollment :

30 participants

Observational Model:

Case-Only

Time Perspective:

Prospective

Official Title:

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

Study Start Date :

Jun 1, 2014

Anticipated Primary Completion Date :

Jun 1, 2016

Anticipated Study Completion Date :

Jun 1, 2017

Outcome Measures

Primary Outcome Measures

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

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Month to 21 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	CHU Sainte-Justine	Montréal	Quebec	Canada	H3T1C5

Sponsors and Collaborators

St. Justine's Hospital

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Dr Monia Marzouki, Hematologist/Oncologist, St. Justine's Hospital

ClinicalTrials.gov Identifier:

NCT02242552

Other Study ID Numbers:

Triceps

First Posted:

Sep 17, 2014

Last Update Posted:

Sep 17, 2014

Last Verified:

Aug 1, 2014

Study Results

No Results Posted as of Sep 17, 2014