A new randomized, double-blind, placebo-controlled, phase 3 clinical trial assessing the safety and efficacy of ropeginterferon alfa-2b, a new-generation pegylated interferon-based therapy, in patients with early and lower-risk primary myelofibrosis (MF) is now open.
The trial aims to recruit 150 such patients who are at least 18 years of age and will receive either up to 500 μg of subcutaneous ropeginterferon alfa-2b or a placebo every 2 weeks until 56 weeks.
The primary endpoints of the trial include clinically relevant complete hematologic response as measured by platelet count, white blood cell count, hemoglobin levels in peripheral blood, absence of thrombotic events, and no progression to acute myeloid leukemia, and symptom endpoint.
Secondary endpoints include bone marrow response, event-free survival or progression-free survival, molecular response in driver or relevant coexisting gene mutations, and safety.
“The study will provide important data for the treatment of early/lower-risk [primary] MF for which an anti-clonal, disease-modifying agent is highly needed,” the researchers wrote in an article that they published in the journal Annals of Hematology, which contains the details of the trial design.
The trial is not yet recruiting participants. It is estimated to start in October 2024 and be completed in August 2027.
Previous research has shown that ropeginterferon alfa-2b has favorable pharmacokinetics and safety profiles and requires less frequent injections than previous formulations of pegylated interferon alfa, the researchers noted.