This pilot phase II trial studies how well selumetinib works in treating patients with
neurofibromatosis type 1 and cutaneous neurofibromas. Selumetinib may stop the growth of
tumor cells by blocking some of the enzymes needed for cell growth.
I. Determine if selumetinib can result in shrinkage of cutaneous neurofibromas.
I. Assess the effect of selumetinib on target inhibition in cutaneous neurofibroma(s) excised
prior treatment and on treatment with selumetinib for analysis of percent inhibition of
phosphorylated ERK (pERK), and changes in phosphorylated AKT (pAKT).
I. Assess the effect of selumetinib on the development on new cutaneous neurofibromas while
on treatment with selumetinib.
II. Assess the effect of selumetinib on target inhibition in cutaneous neurofibroma(s)
excised prior treatment and on treatment with selumetinib for analysis of the tumor kinome.
III. Assess the effect of selumetinib skin related morbidity and pain using the Skindex, the
Global Impression of Change Scale and Numeric Rating Scale, all of which are patient reported
IV. Quantify the development of new cutaneous neurofibromas on treatment with selumetinib.
V. Detailed pathologic analysis of cutaneous neurofibromas pretreatment and on treatment with
selumetinib for changes in cell composition (including macrophage and mast cell
VI. Investigate alterations that correlate with cutaneous neurofibroma (cNF) response to
selumetinib treatment with pilot genomic, deoxyribonucleic acid (DNA) methylation, and
Patients receive selumetinib orally (PO) twice daily (BID) on days 1-28. Treatment repeats
every 28 days for up to 24 cycles in the absence of disease progression or unacceptable
toxicity. Patients who experience a volume decrease in the target cutaneous neurofibromas may
continue treatment for 12 additional cycles.
After completion of study treatment, patients are followed up every 4 months for 1 year.
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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