Phase 2 Study of Imatinib in Children with Neurofibromatosis and Airway Tumors

K
Kent Robertson, MD

Primary Investigator

Overview

The purpose of this study is to look at a subset of plexiform neurofibromas and determine if the airway tumors are more sensitive to imatinib therapy.

Description

The purpose of this study is to determine if NF1 associated pediatric airway-related plexiform neurofibromas represent a subset of plexiform neurofibromas with an inherent sensitivity to imatinib therapy and if imatinib therapy will show efficacy by improving respiratory function as assessed by pulmonary function tests and sleep study evaluations as children with airway tumors.

Eligibility

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    neurofibromas
  • Age: Between 1 Years - 12 Years
  • Gender: All

Inclusion Criteria
Diagnosis of neurofibromatosis type 1 (NF1).
Presence of symptomatic airway plexiform neurofibromas ; defined by abnormal sleep study or pulmonary function testing
Adequate end organ function
Patients must be able to swallow whole pills or crushed pills in a soft food such as pudding or apple sauce; or have other GI access such as a G-tube
Exclusion Criteria
Patient has received any other investigational agents within 14 days of first day of study drug dosing.
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)
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 requiring therapeutic intervention.
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.
Patient previously received radiotherapy to 25 % of the bone marrow
Patient had a major surgery within 2 weeks prior to study entry.
Patient/parent 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.
Patient has an unstable airway requiring more urgent intervention or deemed unable to travel due to unstable airway by referring MD.


Additional Information:
Participants will be paid for their participation.

Updated on 05 Apr 2023. Study ID: 1505569560

Interested in the study?

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