Study of Imatinib in Children With Neurofibromatosis and Airway Tumors

Withdrawn before enrolling · Phase 2

Conditions studied: Neurofibroma, Plexiform

In brief

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

Key facts

Study ID

NCT03688568

Run by

Indiana University

People needed

0

Starts

2018-09-01

Expected to finish

2021-09-01

Last updated by the study team

2021-02-12

Who can join

Age: 1 and older, up to 12. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Riley Hospital for Children - Indiana University — Indianapolis, Indiana, United States

Full record on ClinicalTrials.gov

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