Phase 12a Study of Mirdametinib for Adults with Neurofibromatosis 1 and Cutaneous Neurofibromas Evaluating Safety, Dosage, and Treatment Effects

What this Trial Is About

Researchers are evaluating mirdametinib monotherapy in adults with Neurofibromatosis 1 NF1 and cutaneous neurofibromas cNF. This Phase 12a, open-label, non-randomized study aims to find safe and effective doses of mirdametinib and assess its anti-tumor activity. The study is conducted at Johns Hopkins University and includes adults with NF1 and measurable cNF who seek systemic treatment due to symptoms like disfigurement, pain, or itching. The study has two phases. Phase 1 tests up to four different dose regimens of mirdametinib, given continuously or intermittently 3 weeks on, 1 week off in 28-day treatment cycles to determine recommended doses for Phase 2a. Phase 2a evaluates the safety and effectiveness of up to two selected doses. Participants receive the study drug until stopping criteria such as treatment completion, disease progression, intolerance, or withdrawal are met. Treatment periods end with the last dose, followed by 30 days of study completion monitoring. Participants undergo screening, treatment, and a post-study safety follow-up at the specialty center. They will be monitored through clinical assessments, laboratory tests, tumor biopsies, and performance evaluations. The main outcomes measured include mirdametinib safety, determination of recommended doses, and preliminary anti-tumor effects. The study continues until participants stop treatment due to various reasons or complete up to 48 treatment cycles.

Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).