Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01140360
• Date of registration: 08/06/2010
• Prospective Registration: Yes
• Primary sponsor: Kent Robertson
• Public title: Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas 0908-09
• Scientific title: Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas
• Date of first enrolment: February 2012
• Target sample size: 21
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01140360
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Kent Robertson, MD PhD
• Affiliation: Indiana University

Key inclusion & exclusion criteria

Inclusion criteria

1. Patients > 3 years of age.

2. Diagnosis of neurofibromatosis type 1 (NF1).

3. Presence of clinically significant plexiform neurofibromas (biopsy proven if possible
with tissue blocks available); defined as 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).

5. Patients must have a Karnofsky of > 70% or Lansky of > 50% and a life expectancy of >
2 months.

6. 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.

7. Patients must be able to swallow whole pills.

8. 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.

9. 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 or equal to 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.

13. Patients who have or anticipate receiving permanent (or semi-permanent) metallic
structures attached to their body. (e.g., braces on teeth, body piercings), which
their physicians believe will interfere with the MRI.



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

Health Condition(s) or Problem(s) studied

Neurofibromatosis

Neurofibromas

Intervention(s)

Drug: Gleevec

Primary Outcome(s)

Disease Response [Time Frame: 1 year]

Secondary ID(s)

0908-09

NF/Gleevec DOD Trial

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 02/07/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01140360