NCT02839720

Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma

Pilot Study of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) for Adults With Neurofibromatosis Type 1 (NF1) and Cutaneous Neurofibromas (CNF)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Cutaneous Neurofibroma
• Neurofibromatosis Type 1
• Optic Nerve Glioma

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Selumetinib Sulfate	Given PO

Timeline

• Start date: 2017-08-26
• Primary completion: 2022-11-05
• Completion: 2023-08-31
• First posted: 2016-07-21
• Last updated: 2023-12-06
• Results posted: 2023-12-05

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study