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

Phase 2
3 Years
65 Years
Not Enrolling

Thank you

Trial Information

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

This is an open-label Phase II Study to determine the efficacy of Gleevec® in
neurofibromatosis (NF1) patients with plexiform neurofibromas with the secondary goals of
determining the toxicity, and tumor markers in this genetically defined population. The
rationale for this study arises from the response of human and murine NF1 cells to Gleevec®
in vitro and the response of a single NF1 patient treated with Gleevec® for airway
compression by a plexiform neurofibroma with a dramatic response not previously seen in NF1
therapy. The plan of therapy will include oral dosing of Gleevec® at 440 mg/m2/day (max 800
mg/day) for pediatric subjects and 800 mg/day for adult patients. (with 25% dose reduction
for significant toxicity). Treatment will continue for 6 months with an option to continue
as long as the patient remains on study provided the patient shows benefit from treatments
with Gleevec® and there are no safety concerns.

Inclusion Criteria:

1. Patients 3-65 years of age.

2. Diagnosis of neurofibromatosis (NF1), as outpatients.

3. Presence of clinically significant plexiform neurofibromas (biopsy proven if possible
with tissue blocks available); that is tumors that are potentially life threatening
or are impinging on vital structures or significantly impair the quality of life from
pain or other symptoms.

4. Patients must have measurable disease by magnetic resonance imaging (MRI). Patients
must have a Karnofsky or Lansky Performance score of > 80% and a life expectancy of
> 2 months.

5. Adequate end organ function, defined as the following:

total bilirubin < 1.5 x ULN, SGOT and SGPT < 2.5 x UNL, creatinine < 1.5 x ULN, ANC >
1.5 x 109/L, platelets > 100 x 109/L.

6. Female patients of childbearing potential must have negative pregnancy test within 7
days before initiation of study drug dosing. Postmenopausal women must be amenorrheic
for at least 12 months to be considered of non-childbearing potential. Male and
female patients of reproductive potential must agree to employ an effective barrier
method of birth control throughout the study and for up to 3 months following
discontinuation of study drug.

7. Written, voluntary informed consent.

Exclusion criteria:

1. Patient has received any other investigational agents within 28 days of first day of
study drug dosing, unless the disease is rapidly progressing.

2. Patient is < 5 years free of another primary malignancy except: if the other primary
malignancy is not currently clinically significant nor requiring active intervention,
or if other primary malignancy is a basal cell skin cancer or a cervical carcinoma in
situ. Existence of any other malignant disease is not allowed.

3. Patient with Grade III/IV cardiac problems as defined by the New York Heart
Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6
months of study)

4. Female patients who are pregnant or breast-feeding.

5. Patient has a severe and/or uncontrolled medical disease (i.e., uncontrolled
diabetes, chronic renal disease, or active uncontrolled infection).

6. Patient has a known brain metastasis. Non-specific CNS changes on MRI/CT
characteristic of NF1 are allowed, but not known CNS malignancies.

7. Patient has known chronic liver disease (i.e., chronic active hepatitis, and

8. Patient has a known diagnosis of human immunodeficiency virus (HIV) infection.

9. Patient received chemotherapy within 4 weeks (6 weeks for nitrosourea or mitomycin-C)
prior to study entry, unless the disease is rapidly progressing.

10. Patient previously received radiotherapy to greater than 25 % of the bone marrow

11. Patient had a major surgery within 2 weeks prior to study entry.

12. Patient with any significant history of non-compliance to medical regimens or with
inability to grant reliable informed consent.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Tumor Response

Outcome Description:

Tumor response will be evaluated using the RECIST criteria.

Outcome Time Frame:

6 months

Safety Issue:



United States: Food and Drug Administration

Study ID:




Start Date:

May 2006

Completion Date:

August 2012

Related Keywords:

  • Neurofibromatosis
  • Gleevec
  • Neurofibroma
  • Neurofibromatoses
  • Neurofibromatosis 1
  • Osteitis Fibrosa Cystica
  • Neurofibroma, Plexiform



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