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 the 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- 3 ½ Tablespoons of blood (depending on your weight) will be 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 to > 25% of the bone marrow

4. Patient had major surgery 2 weeks prior to study entry

5. Poor health status (ie. 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

Indianapolis, Indiana

WHOM TO CONTACT WITH QUESTIONS ABOUT THIS TRIAL

Shannon Burke
Study Coordinator
Phone: 317-278-3395
Email: slbottor@iupui.edu