A Phase 3, Randomized, Double-blind Study of Navtemadlin Plus Ruxolitinib Versus Placebo Plus Ruxolitinib in JAK Inhibitor-Naive Patients With Myelofibrosis With Suboptimal Response to Ruxolitinib

What this Trial Is About

Researchers are studying whether adding navtemadlin to ruxolitinib treatment can provide more benefit than ruxolitinib alone for adults with Myelofibrosis who have not responded well to ruxolitinib by itself. This Phase 3 trial includes patients who are new to JAK inhibitor treatment and have a confirmed diagnosis of primary or post-polycythemia vera or post-essential thrombocythemia Myelofibrosis. The study aims to assess improvements in spleen size and symptom reduction over 24 weeks, as well as long-term outcomes like disease progression and overall survival. Participants first receive ruxolitinib alone during a run-in period to identify those with suboptimal response. Those qualifying are randomly assigned in a 21 ratio to receive either navtemadlin or a placebo as an add-on to their ongoing ruxolitinib. Navtemadlin or placebo is taken orally once daily for 7 days followed by 21 days off in 28-day cycles, while ruxolitinib is taken twice daily continuously. The study is double-blinded so neither participants nor researchers know which add-on treatment is given. During the trial, participants will undergo evaluations including spleen volume measurements and symptom assessments at 24 weeks. They will be monitored for disease progression and survival for up to 8 years. The study includes regular check-ups to track side effects, treatment adherence, and overall health status. These detailed assessments help researchers understand the potential benefits and safety of adding navtemadlin for patients with Myelofibrosis.

Study of Navtemadlin add-on to Ruxolitinib in JAK Inhibitor-Naïve Patients With Myelofibrosis Who Have a Suboptimal Response to Ruxolitinib