Phase III Study of Roginolisib Combined With Ruxolitinib for Adults With Myelofibrosis Unresponsive to JAK Inhibitors

What this Trial Is About

Researchers are investigating how the drug roginolisib works when combined with the standard treatment ruxolitinib in adults with myelofibrosis MF who have not responded well to JAK inhibitors. This Phase III study focuses on evaluating the safety and tolerability of this combination therapy in patients who have been on ruxolitinib for at least three months without significant reduction of spleen size, aiming to better understand the benefit and risk profile. Participants will receive roginolisib at a dose of 80 mg along with ruxolitinib up to 25 mg twice daily. The study is conducted in two parts the first part enrolls 13 patients to assess initial safety, and the second part enrolls an additional 13 patients to further evaluate the treatment combination. Treatment cycles last 28 days, and participants are expected to remain on treatment for up to 52 weeks. During the study, participants will undergo regular safety assessments including monitoring for adverse events, ECG recordings, laboratory tests, and blood pressure measurements at specific points in each treatment cycle. Researchers will also evaluate biomarker responses, spleen size reduction, symptom improvement using the Myelofibrosis Symptom Assessment Form, and pharmacokinetics of roginolisib. Follow-up for overall survival continues every 12 weeks for up to 96 weeks after the last patient is enrolled.

A Study of Roginolisib in Combination With Ruxolitinib in Patients With Myelofibrosis (MF) Who Are Unresponsive to JAK Inhibitors