New Clinical Trial Offers Hope For Adults With NF1 And CNF

Author: NTAP - NF Research Accelerated

Published On: 10/03/25

Every person living with neurofibromatosis type 1 (NF1) has a story—and nearly all of those stories include cutaneous neurofibromas (cNFs). These benign tumors of the skin may not pose a life-threatening risk, but for the 98% of people with NF1 who develop them, the impact can be profound: socially, emotionally, physically, and functionally.

A new clinical trial designed in collaboration between NTAP, academic, and industry partners aims to change the outlook for people living with cNF. The study is testing whether mirdametinib, a targeted therapy recently approved by the FDA for plexiform neurofibromas, can also offer relief from the debilitating burden of cutaneous tumors in adults. “We know that people with neurofibromatosis type 1 develop cutaneous neurofibromas in about 98% of cases, and despite being histologically benign tumors, they can seriously affect quality of life,” Dr. Carlos Romo, principal investigator of the study at the Johns Hopkins Hospital explained. “Neurofibromas can be painful, itchy, and disfiguring. Studies show that patients often cite them as the most bothersome manifestation of NF1. The search for treatments demands that we take that patient experience very seriously.”

Historical Collaboration Between Industry and Academia to Develop Better Treatments for cNFs

The study, titled “Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and cNF,” launched in February 2024. It is an open-label Phase 1/2a clinical trial designed to assess the feasibility, safety, and efficacy of using mirdametinib to treat cNFs in adults with NF1. Mirdametinib, developed by SpringWorks Therapeutics, is an oral MEK inhibitor that in February 2025 received FDA approval for NF1-associated plexiform neurofibromas. This NTAP-led clinical trial with SpringWorks collaboration, through clinical trial oversight and provision of drug, is evaluating whether it may also benefit adults with cNF. 

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