Clinical Trial: Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Phase II Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform Neurofibromas
Brief Summary:
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.
Detailed Summary:
Clinical objectives
- 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 ¸
Pharmacological study
- 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
Sponsor: St. Justine's Hospital
Current Primary Outcome: Demonstrate the clinical benefit of imatinib in a pediatric patient population with progressing and metabolically active plexiform NFs [ Time Frame: 12 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Changes in NF1 biomarkers after treatment with imatinib [ Time Frame: 12 months ]
Original Secondary Outcome: Same as current
Information By: St. Justine's Hospital
Dates:
Date Received: June 25, 2014
Date Started: June 2014
Date Completion: December 2020
Last Updated: May 5, 2017
Last Verified: May 2017