A phase 2, randomized, open-label, multicenter study to evaluate safety and efficacy of single agent selinexor versus treatment of physician’s choice in patients with previously treated myelofibrosis.

Myelofibrosis is an uncommon condition where the bone marrow, the spongy centre of the bones where blood cells are made, becomes scarred. Because the bone marrow cannot work properly, another organ called the spleen tries to help by making blood cells. This causes the spleen to swell significantly, leading to abdominal pain, bloating, and severe tiredness. Many people also develop anaemia, which means having low levels of red blood cells.

While treatments exist, they do not work for everyone, and over time they can stop working effectively. This clinical trial is testing a targeted medicine called selinexor. Researchers want to compare selinexor directly against standard treatment options that a specialist doctor would normally choose. The goal is to see if selinexor provides a better, safer way to manage the disease.

The main measure of success in this study is whether the medicine can shrink the spleen by at least 35%. The trial team will also look at whether it reduces everyday symptoms—such as fatigue, bone pain, and night sweats—by at least half, and whether it improves red blood cell counts so patients feel stronger and more energetic.

Finding new treatments is vital for people living with myelofibrosis. If selinexor proves effective, it could become an important new option for patients whose previous treatments have stopped working.