Phase 2 Study of Imatinib in Children with Neurofibromatosis and Airway Tumors
Investigation of Investigational Medication Therapy for Airway Plexiform Neurofibromas in Children with NF1
Brief description of study.
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.
Detailed description of study
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 of study
You may be eligible for this study if you meet the following criteria:
- Conditions: neurofibromas
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Age: 1 years - 12 years
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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.
This study investigates the effects of imatinib therapy on children with neurofibromatosis type 1 (NF1) who have airway-related plexiform neurofibromas. Plexiform neurofibromas are a type of tumor that can grow in various parts of the body, including the airway. The study aims to see if these specific tumors are more sensitive to imatinib, which is a type of treatment, and if it can improve breathing problems in these children.
Participants in the study will undergo tests to measure their lung function and sleep quality. These tests will help determine if the imatinib therapy is effective in improving their respiratory health. Complex terms like 'pulmonary function tests' are used to describe tests that check how well the lungs are working. Sleep studies evaluate how well someone sleeps and if they have any breathing problems during sleep.
- Who can participate: Children with neurofibromatosis type 1 (NF1) and symptomatic airway plexiform neurofibromas, as shown by abnormal sleep or lung function tests, can participate. They must be able to take medication orally or through a feeding tube and have adequate organ function.
- Study details: Participants will be required to take an investigational medication, imatinib, and will have their respiratory function monitored through specific tests. A placebo, which is an inactive substance that looks like the investigational medication but contains no medicine, is not mentioned in this study.
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