Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood

NCT02242552 | Unknown DMC

• 1 other identifier: Triceps
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

Pediatric Cancer

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• 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

• Age: 1 Month - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Children and adolescents with Relapse or refractory leukemia and solid tumor

You may qualify if:

• At the time of enrollment:
• 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

You may not qualify if:

• Estimated life expectancy of less than 3 months.

Sponsors & Collaborators

• St. Justine's Hospital: lead

Study Sites (1)

CHU Sainte-Justine

Montreal, Quebec, H3T1C5, Canada

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Blood, saliva, tumor sample (bone marrow or solid tumors)

MeSH Terms

Conditions

Neoplasms

Study Officials

• Monia Marzouki, MD

  St. Justine's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Monia Marzouki, MD

CONTACT

514-345-4931; monia.marzouki@umontreal.ca

Hemrique Bittencourt, MD

CONTACT

514-345-4931; henrique.bittencourt@umontreal.ca

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Hematologist/Oncologist

Study Record Dates

• First Submitted: August 15, 2014
• First Posted: September 17, 2014
• Study Start: June 1, 2014
• Primary Completion: June 1, 2016
• Study Completion: June 1, 2017
• Last Updated: September 17, 2014

Record last verified: 2014-08

Locations

CA (1)