Dr. Hoffman on the Design of an Ongoing Trial Testing PTG-300 in Polycythemia Vera

Ronald Hoffman, MD, a professor of medicine, hematology, and medical oncology at Mount Sinai, discusses the design of an ongoing phase 2 trial (NCT04057040) evaluating the first-in-class synthetic hepcidin mimetic PTG-300 in patients with polycythemia vera. 

According to Hoffman, there are 3 components to the trial. The dose-finding component will determine the optimal dose of PTG-300 that can maintain a patient’s hematocrit levels below 45%, says Hoffman.

Once the optimal dose has been established, patients will be randomized to continue on treatment with PTG-300 or placebo, Hoffman explains. The goal of this portion of the study is to determine whether erythrocytosis will return in patients who are receiving placebo.

Finally, patients randomized to placebo will receive PTG-300 in the continuation phase of the study, Hoffman says. As the frequency of phlebotomy is a metric that is being used to determine the efficacy of PTG-300, eligible patients have to have had more than 3 phlebotomies over a period of 6 months prior to study entry, concludes Hoffman.