NCT06159166

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

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).

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Johns Hopkins University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• NF1
• Cutaneous Neurofibroma
• Monotherapy

Interventions

Type	Name	Description
DRUG	Mirdametinib	This study is designed to assess Mirdametinib safety and efficacy in two phases: phase 1 tests the safety of up to four dose regimens of Mirdametinib, administered continuously or intermittently (3 weeks on/1 week off) to identify up to two recommended phase 2 doses. Phase 2a of the study will test the safety and efficacy of the recommended phase 2 dose(s) in adults with NF1 and cNF. The following dosing strategy will be assessed in participants ≥ 18 years old with NF1 and a minimum of 12 measurable cNF who desire systemic treatment of their cNF due to disfigurement, pain or itching. Each treatment cycle in this study is 28 days.treatment cycle in this study is 28 days.

Timeline

• Start date: 2024-02-12
• Primary completion: 2027-11-15
• Completion: 2028-11-15
• First posted: 2023-12-06
• Last updated: 2026-04-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study