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

Indiana University1 site in 1 countrySeptember 1, 2018

ConditionsNeurofibroma, Plexiform

InterventionsImatinib Mesylate

DrugsImatinib Mesylate

Overview

Phase: Phase 2
Intervention: Imatinib Mesylate
Conditions: Neurofibroma, Plexiform
Sponsor: Indiana University
Locations: 1
Primary Endpoint: Quantitative Functional Airway Response
Status: Withdrawn
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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. Funding Source - FDA OOPD

Registry

clinicaltrials.gov

Start Date

September 1, 2018

End Date

September 1, 2021

Last Updated

5 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Indiana University

Responsible Party

Principal Investigator

Kent A Robertson

Associate Professor

Indiana University

Eligibility Criteria

Inclusion Criteria

•Patients aged: \> 6 months and \< 12 years of age.
•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.
•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 or crushed pills in a soft food such as pudding or apple sauce; or have other GI access such as a 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 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.

Arms & Interventions

Imatinib Mesylate Arm

Imatinib Mesylate, given daily orally, 55 mg PO BID, if tolerated for 2 weeks increase to 110 mg/m2 BID, and further increase to 165 and final dosage to 220 mg/m2 bid if tolerated. Can continue for 12 months.

Intervention: Imatinib Mesylate