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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01673009
• Date of registration: 22/08/2012
• Prospective Registration: No
• Primary sponsor: Indiana University
• Public title: Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas
• Scientific title: Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas
• Date of first enrolment: May 2006
• Target sample size: 36
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01673009
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

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
cirrhosis).

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.



Age minimum: 3 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Neurofibromatosis

Intervention(s)

Drug: Gleevec

Primary Outcome(s)

Percent Change From Baseline in Tumor Volume at 6 Months [Time Frame: baseline to 6 months]

Secondary Outcome(s)

Serum Bioactivity [Time Frame: 7 days and 1 month]

Secondary ID(s)

0512-25

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 06/04/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01673009