Selumetinib in Treating Patients With Neurofibromatosis Type 1 & Cutaneous

Status Completed

Clinical Trial Summary

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.

Clinical Trial Description

PRIMARY OBJECTIVE: I. Determine if selumetinib sulfate (selumetinib) can result in shrinkage of cutaneous neurofibromas. SECONDARY OBJECTIVE: 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). EXPLORATORY OBJECTIVES: 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 outcome measures. 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 infiltration). VI. Investigate alterations that correlate with cutaneous neurofibroma (cNF) response to selumetinib treatment with pilot genomic, deoxyribonucleic acid (DNA) methylation, and transcriptomic studies. OUTLINE: Patients receive selumetinib sulfate 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT02839720
Study type	Interventional
Source	National Cancer Institute (NCI)
Contact
Status	Completed
Phase	Phase 2
Start date	August 26, 2017
Completion date	August 31, 2023

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See also
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Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms