Gleevec NF1 Trial

    

New Experimental Trial for Treating NF

 

Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patients with Plexiform Neurofibromas

STUDY PURPOSE

The purpose of this study is to find out if the drug, Gleevec, is safe and effective in treating plexiform neurofibromas.  Gleevec interferes with certain cancer cell molecules involved in the growth and multiplication of cancer cells.  It is suspected that Gleevec may have the same effect on plexiform neurofibromas, shrinking or stopping the tumor growth.

 
STUDY PARTICIPATION

Gleevec is taken by mouth for up to 6 months depending on the benefit from the treatment and no safety concerns.  Participants will have approximately 1 to 3.5 Tablespoons of blood (depending on your weight) drawn for research purposes at 3 different time points while participating in the study.  MRI scans will be performed to assess the response to this therapy.  These scans are required prior to starting this treatment, after 2, 6, 12 and 24 months of treatment.  Physical exams and blood work will also be required to monitor your overall health status while participating in this study.

 

STUDY TYPE

This study is considered investigational.

 

WHO IS ABLE TO PARTICIPATE

Inclusion criteria

  1. Patients 3-65 years of age
  2. Diagnosis of a clinically significant plexiform neurofibromas
  3. Overall good health status
  4. Female patients must not be pregnant
 

Exclusion criteria

  1. Patient has received any other investigational drugs within 28 days of starting this therapy
  2. Patient received chemotherapy within 4 weeks prior to starting this therapy
  3. Patient received radiotherapy equal or greater than 25% of the bone marrow
  4. Patient had major surgery 2 weeks prior to study entry
  5. Poor health status (i.e. Some chronic illnesses, significant cardiac abnormalities)
 

EXPECTED NUMBER OF PATIENTS THAT WILL PARTICIPATE

This study will accrue 40 patients total:

 

Pediatric Patients

        10 pediatric patients between 3 and 9 years of age

        10 pediatric patients between 10 and 18 years of age

 

Adult Patients

        20 adult patients between 19 and 65 years of age

 

WHERE IS THIS TRIAL BEING CONDUCTED

Indiana University Medical Center / Riley Hospital in Indianapolis, Indiana

 

WHO TO CONTACT WITH QUESTIONS ABOUT THIS TRIAL

Mary Heskett, RN

Clinical Research Nurse

Phone: 317-274-2832

 

Kent Robertson, MD, PhD

Principle Investigator

Phone: 317-278-9877