Myelofibrosis

This phase II trial compares the effect of ASTX727 in combination with iadademstat to ASTX727 alone in treating patients with accelerated or blast phase Philadelphia chromosome negative myeloproliferative neoplasms (MPNs). ASTX727 is a combination of two drugs, cedazuridine and decitabine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Iadademstat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with iadademstat may be more effective than ASTX727 alone in treating patients with accelerated or blast phase Philadelphia chromosome negative MPNs.

In transfusion-dependent myelodysplastic syndromes patients, regular blood transfusions lead to iron overload, which can cause organ damage, hormonal imbalances, and increased infection risk, ultimately impacting patient survival. Standard oral iron chelation therapies can be intolerable for some patients due to adverse effects. The MEX-CD1 device (class III) could potentially offer an alternative for these patients by reducing serum iron levels through a novel, extracorporeal approach.
MEXIRON clinical investigation focuses on the use of MEX-CD1, a medical device designed for extracorporeal chelation therapy to reduce serum iron overload in patients suffering from transfusion-dependent myelodysplastic syndromes (MDS) and myelofibrosis.
MEXIRON aims to evaluate the device's use feasibility, safety, and effectiveness in reducing serum free iron levels. Transfusions needs, patient experience and quality of life are also assessed.
Each enrolled patients will undergo three low-volume continuous veno-venous haemodialysis cycles within one week.
Following the three- haemodialysis cycles, patients will be monitored through on-site follow-up visits at 7 days, 28 days, and 90 days post-treatment to assess long-term effects.

To assess INCB05643 + ruxolitinib JAKi-naive patients with myelofibrosis