Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma

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.

Timeline

Start

2017-08-26

Primary completion

2022-11-05

Completion

2023-08-31

Publications

• Gross AM, Reid OH, Baldwin LA, Cannon A, Choo-Wosoba H, Steinberg SM, Lobbous M, Wolters PL, Pichard DC, Tibery CM, Dombi E, Derdak J, Widemann BC, Korf BR. Treatment of Cutaneous Neurofibromas in Neurofibromatosis Type 1 With MEK Inhibitor Selumetinib: A Nonrandomized Clinical Trial. JAMA Dermatol. 2025 Feb 26;161(5):533-7. doi: 10.1001/jamadermatol.2024.6574. Online ahead of print.
• Church C, Fay CX, Kriukov E, Liu H, Cannon A, Baldwin LA, Crossman DK, Korf B, Wallace MR, Gross AM, Widemann BC, Kesterson RA, Baranov P, Wallis D. snRNA-seq of human cutaneous neurofibromas before and after selumetinib treatment implicates role of altered Schwann cell states, inter-cellular signaling, and extracellular matrix in treatment response. Acta Neuropathol Commun. 2024 Jun 21;12(1):102. doi: 10.1186/s40478-024-01821-z.

Drugs

Indications