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Personalized Targeted Therapy in Refractory or Relapsed Cancer in Childhood

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02242552
Recruitment Status : Unknown
Verified August 2014 by Dr Monia Marzouki, St. Justine's Hospital.
Recruitment status was:  Recruiting
First Posted : September 17, 2014
Last Update Posted : September 17, 2014
Information provided by (Responsible Party):
Dr Monia Marzouki, 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.

Condition or disease
Pediatric Cancer

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Study Type : Observational
Estimated 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 : June 2014
Estimated Primary Completion Date : June 2016
Estimated Study Completion Date : June 2017

Primary Outcome Measures :
  1. Feasibility of performing genomic data-based targeted therapy clinical trials in childhood cancers with poor prognosis, including relapsed or refractory cancers. [ Time Frame: 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 :
  1. Number of children with cancer who are suitable candidates for targeted therapy at our institution each year. [ Time Frame: 24 months ]
  2. Number and type of driver mutation(s) found in our population of recurrent or refractory cancers. [ Time Frame: 24 months ]
  3. Number of cancer patients who harbour actionable driver mutation(s) that can be targeted with a Health Canada approved targeted drug. [ Time Frame: 24 months ]
  4. 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 [ Time Frame: 24 months ]

Biospecimen Retention:   Samples With DNA
Blood, saliva, tumor sample (bone marrow or solid tumors)

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Month to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Children and adolescents with Relapse or refractory leukemia and solid tumor

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02242552

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Contact: Monia Marzouki, MD 514-345-4931 ext 6316
Contact: Hemrique Bittencourt, MD 514-345-4931 ext 2724

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Canada, Quebec
CHU Sainte-Justine Recruiting
Montréal, Quebec, Canada, H3T1C5
Contact: Dominique Lafrenière, BScN, DESS    514-345-4969   
Contact: Mary-Ellen French, RN    514-345-4969   
Sponsors and Collaborators
St. Justine's Hospital
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Principal Investigator: Monia Marzouki, MD St. Justine's Hospital
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Responsible Party: Dr Monia Marzouki, Hematologist/Oncologist, St. Justine's Hospital Identifier: NCT02242552    
Other Study ID Numbers: Triceps
First Posted: September 17, 2014    Key Record Dates
Last Update Posted: September 17, 2014
Last Verified: August 2014