NF1 Clinical Trial
Official title:
A Phase 1/2a Open-label, Multicenter Dose Finding Study to Evaluate the Safety and Anti-tumor Activity of Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
This is a Phase 1/2a, open-label, non-randomized, multi-dose study of mirdametinib monotherapy in adults with NF1 and cNF. In both Phases of the study, participation in the study will comprise three periods: screening, treatment and post-study safety follow-up to be performed at the NF1 and cNF specialty center: Johns Hopkins University.
This is a Phase 1/2a, open-label, non-randomized, multi-dose study of mirdametinib monotherapy in adults with NF1 and cNF. The study will be conducted in two phases: Phase 1 will test the safety of multiple dose regimens of mirdametinib, in order to identify a recommended phase 2 dose(s) for phase 2a. The phase 1 portion will enroll participants in dose regimens 1 to 3 concurrently. Enrollment of participants to dose regimen 4 will be conducted only if no dose limiting toxicities (DLT) occurred in any of the three previous regimens within at least, the first three cycles. The study will continue until one of the following stopping conditions is met. - The RP2D(s) have been identified with sufficient accuracy. The dose regimen(s) selected as the RP2D have been fully enrolled with at least 3 cycles of treatment completed for all participants) - All dose regimens are deemed to have unacceptable safety Phase 2a of the study will test the efficacy and safety of a maximum of two RP2Ds in adults with NF1 and cNF. This study will be considered complete once all participants have ceased study treatment due to death, disease progression, intolerance, withdrawal of consent from the study, physician discretion, or completed a maximum of 48 cycles of treatment across both phase 1 and phase 2a. Each treatment cycle is 28 days. In both Phases of the study, participation in the study will comprise three periods: screening, treatment and post-study safety follow-up to be performed at Johns Hopkins University. ;
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