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Phase II clinical research study with patients suffering from neurofibromatosis type 1 and progressive plexiform neurofibroma

Invitation to take part in this fundamental and clinical research study

CHU Sainte-Justine has set up a team of multidisciplinary clinical and fundamental researchers to assess the effects of a new treatment for children and young adults suffering from neurofibromatosis type 1, with one or more progressive plexiform neurofibromas.

Nature of the project

Other than surgery, there are presently no other standard medical treatments for secondary plexiform neurofibromas of neurofibromatosis type 1.

The objective of the study is to assess whether taking medication daily (imatinib mesylate) can limit or halt the progression of this type of tumor.

Imatinib mesylate is successfully used with patients suffering from different types of leukemia and cancers. Imatinib is part of the targeted therapies and its therapeutic action is associated with its binding to specific cellular receptors involved in the proliferation of tissues. Imatinib has proven that it can bind to a cellular receptor involved in the growth of plexiform neurofibromas (c-KIT receptor).

This Phase II study intends to recruit 25 assessable patients with NF1 and progressive PN. The entire study for each of these patients is one year or less depending on the patient’s response to treatment. The medication (available commercially) will be given free of charge to all participants for the total duration of the study.

The project comprises one visit before the study begins to discuss the protocol in effect and obtain the patient’s consent. Thereafter, if any additional tests are required, they will be done during the same visit or during a follow-up visit, before the medication is given to the patient. Regular tests with respect to blood count will be performed (usually every month or two) and radiological examinations to evaluate the patient’s response to the treatment will be done every three months over the course of 1 year. These visits will take place at CHU Sainte-Justine’s oncology clinic for the total duration of the study

Inclusion criteria

  • Be between 2 and 21 years of age
  • Be a carrier of neurofibromatosis type 1 and display one or more inoperable plexiform neurofibromas or progressive plexiform neurofibromas that limit some vital functions or that cause irreparable harm to the patient (for example: physical appearance).

Drawbacks and risks

There can be risks associated with taking part in this study. Those risks are discussed in great detail with all interested patients.

Benefits and advantages

This research could potentially lead to arresting or stopping the progression of the progressive plexiform neurofibroma. There are no guarantees on any of the potential benefits or advantages. Taking part in this study under the close supervision of the research team could lead to the development of a medical treatment that may halt the progression of pathologies associated with neurofibromatosis type 1.

Financial compensation

There is no compensation offered for a patient’s participation in this study.

Additional information

If you are interested in participating in this study or would like additional information, please contact:

Members of CHU Sainte-Justine Research Team

Clinical Objectives

  • Haematology
    Dr. Jean-Marie Leclerc, Dr. Monia Marzouki
  • Neurology
    Dr. Albert Larbrisseau, Dr. Sébastien Perreault, Dr. Claude Mercier
  • Radiology
    Dr. Ramy El-Jalbout
  • Pathology
    Dr. Rose Chami

Biological Study Objectives and Tissue Bank

  • Zoulfia Allakhverdie, PhD
  • Alain Moreau, PhD
  • Dr. Michel Duval
  • Daniel Sinnett, PhD

Objectives for Studies - FDG Petscan

  • Nuclear medicine: Dr. Sophie Turpin, Dr. Raymond Lambert

Pharmacological Studies

  • Yves Théoret, PhD
  • Pharmacist: Lyne Pedneault

Research Coordinator

  • Dominique Lafrenière, BScN

Pivot Nurse

  • Émilie Allard-Villeneuve, BScN

Regulatory Affairs

  • Simon Alexander, Proficio Inc.

Access to imatinib

  • Pharmascience Canada Inc.
About this page
Updated on 11/5/2014
Created on 8/19/2014
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