Imatinib Mesylate in Treating Children with Neurofibromatosis and Airway Tumors

Status: Active


This phase II trial studies how well imatinib mesylate works in treating children with neurofibromatosis and airway tumors. Imatinib mesylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Eligibility Criteria

Inclusion Criteria

  • Diagnosis of neurofibromatosis type 1 (NF1)
  • Presence of symptomatic airway plexiform neurofibromas; defined by abnormal sleep study or pulmonary function testing
  • Patients must have measurable (>= 1.5 cm in two dimensions or able to assess a minimum of 3 slices) disease by magnetic resonance imaging (MRI)
  • Patients must have a Karnofsky of >= 70% or Lansky of >= 50% and a life expectancy of >= 2 months
  • Total bilirubin < 1.5 x upper limit of normal (ULN)
  • Serum glutamic-oxaloacetic transaminase (SGOT) and serum glutamate pyruvate transaminase (SGPT) < 2.5 x UNL
  • Creatinine < 1.5 x ULN
  • Absolute neutrophil count (ANC) > 1.5 x 10^9/L
  • Platelets > 100 x 10^9/L
  • Patients must be able to swallow whole pills or crushed pills in a soft food such as pudding or apple sauce; or have other gastrointestinal (GI) access such as a gastrostomy (G)-tube
  • Written, voluntary informed consent/assent

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 central nervous system (CNS) changes on MRI/computed tomography (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 Doctor of Medicine (MD)

Locations & Contacts


Indiana University / Melvin and Bren Simon Cancer Center
Status: Active
Contact: Kent Allen Robertson
Phone: 317-944-8784

Trial Objectives and Outline


I. Determine quantitative functional airway response via sleep study and pulmonary function tests in pediatric patients with symptomatic airway plexiform neurofibromas after treatment with oral imatinib mesylate (imatinib) by individual dose escalation from 110 to 440 mg/m^2/day for 12 months.

II. Determine the response of airway plexiform tumors compared to non-airway plexiform tumors in the same patients using volumetric magnetic resonance imaging (MRI) size determinations following 12 months of oral imatinib.

III. Perform biomarker and biologic effect assessment with circulating cytokine levels including SCF, TGF- β, PDGF, and midkine; circulating levels of inflammatory monocytes and angiogenic progenitor cells by flow cytometry; collect basic quality of life information and further assess the safety and tolerability of imatinib in a group of patients with neurofibromatosis (NF1) and airway plexiform tumors.


Patients receive imatinib mesylate orally (PO) twice daily (BID) for up to 12 months in the absence of disease progression or unacceptable toxicity.

Trial Phase & Type

Trial Phase

Phase II

Trial Type


Lead Organization

Lead Organization
Indiana University / Melvin and Bren Simon Cancer Center

Principal Investigator
Kent Allen Robertson

Trial IDs

Secondary IDs NCI-2018-02542 ID NCT03688568