Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02177825|
Recruitment Status : Terminated (Slow accrual 5 patients out of 25 expected. Primary objectives could not be met without recruiting more patients.)
First Posted : June 30, 2014
Last Update Posted : April 8, 2019
This phase II trial will test the hypothesis that inhibition of c-kit signalling pathways in pediatric patients with Neurofibromatosis Type I(NF-1) and progressing plexiform neurofibroma will result in objective reduction and/or inhibition of plexiform neurofibromas progression.
This will be a Phase II study of imatinib mesylate given orally. Patients with stable or responding disease may receive the drug for a period not exceeding one year.
|Condition or disease||Intervention/treatment||Phase|
|Plexiform Neurofibromas||Drug: Imatinib Mesylate||Phase 2|
- Demonstrate the clinical benefit of imatinib in a pediatric patient population with progressing and metabolically active plexiform neurofibromas (NF)
- Demonstrate the need or not to pursue treatment for more than a year in responders to imatinib
Biological studies objectives
- Identify biological markers of plexiform neurofibroma progression and response to treatment
- Identify biological markers of mast cell responses to imatinib, given that mast cells are required for tumorigenesis and are a target for imatinib
Imaging studies objectives
Using 18-Fluorodeoxyglucose-positron Emission Tomography (FDG PET/CT):
- Identify imaging characteristics of progressing plexiform neurofibromas
- Assess the role of F18-FDG PET/CT in comparison with CT/MRI to evaluate response to imatinib ¸
- Evaluate trough plasma levels of imatinib and its active metabolite (NDMIL N-desmethyl imatinib) achieved in this pediatric population
- Identify potential correlation between imatinib (and NDMI) trough levels achieved and clinical response
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas|
|Study Start Date :||June 2014|
|Actual Primary Completion Date :||March 1, 2019|
|Actual Study Completion Date :||March 1, 2019|
Experimental: Imatinib Mesylate
Imatinib Mesylate at 110 mg/m2 up to 440mg/m2 PO per day for twelve months taken in one morning dose (if dose is less than 200 mg/day) or two doses (morning and evening)
Drug: Imatinib Mesylate
Other Name: Imatinib
- Demonstrate the clinical benefit of imatinib in a pediatric patient population with progressing and metabolically active plexiform NFs [ Time Frame: 12 months ]Time to tumor progression as assessed by volumetric MRI and FDG-PETScan analysis at baseline, after 3, 6, 9, and 12 months on therapy.
- Changes in NF1 biomarkers after treatment with imatinib [ Time Frame: 12 months ]Patients on imatinib will have blood samples and urine taken at baseline and every 3 months until treatment completion or until disease progression. Mastocyte activation biomarkers will be monitored and evaluated as potential disease state indicators.
- Evaluate trough plasma levels of imatinib and its active metabolite (NDMIL N-desmethyl imatinib) achieved in this pediatric population [ Time Frame: 12 months ]We will evaluate trough plasma levels of imatinib achieved in this patient population at 3, 6, 9 and 12 month and correlate it with response to treatment
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02177825
|Montreal, Quebec, Canada, H3T 1C5|
|Principal Investigator:||Sébastien Perreault, MD/FRCPC||St. Justine's Hospital|