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.
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.
This study is considered investigational.
WHO IS ABLE TO PARTICIPATE
- Patients 3-65 years of age
- Diagnosis of a clinically significant plexiform neurofibromas
- Overall good health status
- Female patients must not be pregnant
- Patient has received any other investigational drugs within 28 days of starting this therapy
- Patient received chemotherapy within 4 weeks prior to starting this therapy
- Patient received radiotherapy equal or greater than 25% of the bone marrow
- Patient had major surgery 2 weeks prior to study entry
- 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:
· 10 pediatric patients between 3 and 9 years of age
· 10 pediatric patients between 10 and 18 years of age
· 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 TRIALMary Heskett, RN
Clinical Research Nurse
Kent Robertson, MD, PhD