Clinical Trials & COVID-19

Study Drug

What is the study drug?

The investigational study drug, mirdametinib (PD-0325901), is a capsule (or dispersible tablet, if needed) taken by mouth. It is known as a MEK 1/2 inhibitor. This means the study drug is designed to help block the activity of 2 proteins, called MEK1 and MEK2, that allow tumor growth in people with Neurofibromatosis type 1 (NF1).

What does "investigational" mean?

The study drug is considered “investigational” because it has not been approved by the United States Food and Drug Administration (FDA) or other regulatory authorities for treating NF1-related tumors. It can only be used in research studies.

Will all study participants receive the study drug?

Yes. Everyone who participates in the ReNeu study will receive the study drug.

Have other people used the study drug?

Yes. The study drug has been used by people in Phase 1 and Phase 2 clinical trials.