Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform Neurofibromas
This is an open-label Phase II Study to determine the efficacy of Gleevec® in
neurofibromatosis (NF1) patients with plexiform neurofibromas with the secondary goals of
determining the toxicity, and tumor markers in this genetically defined population. The
rationale for this study arises from the response of human and murine NF1 cells to Gleevec®
in vitro and the response of a single NF1 patient treated with Gleevec® for airway
compression by a plexiform neurofibroma with a dramatic response not previously seen in NF1
therapy. The plan of therapy will include oral dosing of Gleevec® at 440 mg/m2/day (max 800
mg/day) for pediatric subjects and 800 mg/day for adult patients. (with 25% dose reduction
for significant toxicity). Treatment will continue for 6 months with an option to continue
as long as the patient remains on study provided the patient shows benefit from treatments
with Gleevec® and there are no safety concerns.
Interventional
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Tumor Response
Tumor response will be evaluated using the RECIST criteria.
6 months
No
United States: Food and Drug Administration
0512-25
NCT01673009
May 2006
August 2012
Name | Location |
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Indiana University | Indianapolis, Indiana 46202 |