A Phase III Open-Label Multi-Centre Study to Assess Safety and Tolerability of Roginolisib with Ruxolitinib in Adults with Myelofibrosis Unresponsive to JAK Inhibitors

What this Trial Is About

Researchers are evaluating how the drug roginolisib works when combined with ruxolitinib in adults with myelofibrosis MF who have not responded adequately to prior JAK inhibitor treatments. This Phase III open-label study aims to assess the safety and tolerability of this combination treatment in patients with intermediate or high-risk MF. About 26 male and female patients aged 18 or older, who have been on stable doses of ruxolitinib for at least 3 months without significant spleen size reduction, will participate. Participants will receive roginolisib at a dose of 80 mg equivalent to 72 mg roginolisib alongside ruxolitinib up to 25 mg twice daily. The study starts with 13 patients in Part 1 to evaluate initial safety and potential benefits, followed by 13 more patients in Part 2 to further understand the effects of this combination. The treatment cycles are 28 days long, and the treatment period is expected to last up to 52 weeks. During the study, participants will have regular assessments including monitoring of adverse events, ECGs, laboratory tests, and blood pressure at specific days within each cycle. Researchers will also measure biomarker responses, spleen size reduction, symptom improvements, and drug levels in the blood. Participants will be followed for overall survival every 12 weeks for up to 96 weeks after enrollment of the last patient, with symptom assessments at baseline, 12 and 24 weeks. Safety and tolerability will be closely monitored throughout the study period.

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