Status:
COMPLETED
Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas
Lead Sponsor:
Kent Robertson
Conditions:
Neurofibromatosis
Neurofibromas
Eligibility:
All Genders
3-65 years
Phase:
PHASE1
PHASE2
Brief Summary
This is a second Pilot Study to determine the efficacy of Gleevec® in neurofibromatosis (NF1) patients with plexiform neurofibromas using new response assessment modalities with the secondary goals of...
Eligibility Criteria
Inclusion
- Inclusion criteria
- Patients \> 3 years of age.
- Diagnosis of neurofibromatosis type 1 (NF1).
- 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.
- Patients must have measurable disease by magnetic resonance imaging (MRI).
- Patients must have a Karnofsky of \> 70% or Lansky of \> 50% and a life expectancy of \> 2 months.
- 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.
- Patients must be able to swallow whole pills.
- 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.
- Written, voluntary informed consent.
- Exclusion criteria
- Patient has received any other investigational agents within 28 days of first day of study drug dosing, unless the disease is rapidly progressing.
- 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.
- 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)
- Female patients who are pregnant or breast-feeding.
- Patient has a severe and/or uncontrolled medical disease (i.e., uncontrolled diabetes, chronic renal disease, or active uncontrolled infection).
- Patient has a known brain metastasis. Non-specific CNS changes on MRI/CT characteristic of NF1 are allowed, but not known CNS malignancies.
- Patient has known chronic liver disease (i.e., chronic active hepatitis, and cirrhosis).
- Patient has a known diagnosis of human immunodeficiency virus (HIV) infection.
- Patient received chemotherapy within 4 weeks (6 weeks for nitrosourea or mitomycin-C) prior to study entry, unless the disease is rapidly progressing.
- Patient previously received radiotherapy to greater than or equal to 25 % of the bone marrow
- Patient had a major surgery within 2 weeks prior to study entry.
- Patient with any significant history of non-compliance to medical regimens or with inability to grant reliable informed consent.
- 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.
Exclusion
Key Trial Info
Start Date :
February 1 2012
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2016
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT01140360
Start Date
February 1 2012
End Date
December 1 2016
Last Update
July 2 2017
Active Locations (1)
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1
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202