Phase 2 Study Comparing ASTX727 Alone or With Iadademstat in Adults with Advanced Philadelphia Chromosome-Negative Myeloproliferative Neoplasms

What this Trial Is About

Researchers are studying the treatment of accelerated or blast phase Philadelphia chromosome-negative myeloproliferative neoplasms MPNs, a type of blood cancer, by comparing two drug regimens. This phase II trial evaluates whether combining ASTX727, which includes the drugs cedazuridine and decitabine, with iadademstat is more effective than ASTX727 alone. ASTX727 works by helping bone marrow produce normal blood cells and attacking abnormal ones, while iadademstat may stop tumor growth by blocking enzymes needed for cell growth. Patients are randomly assigned to one of two treatment groups. One group takes ASTX727 orally once daily for five days in a 28-day cycle. The other group takes ASTX727 on the same schedule plus iadademstat orally on days 1-5, 8-12, 15-19, and 22-26 of each 28-day cycle. Treatment cycles continue unless the disease progresses or side effects are unacceptable. During the study, patients provide buccal swabs, blood samples, and bone marrow samples for analysis. Participants are monitored throughout treatment with various tests including blood and bone marrow examinations. After stopping treatment for reasons other than disease progression, patients have follow-up visits every three months if they stop due to progression, follow-up occurs every six months. Researchers measure the rate of complete acute leukemia response within four treatment cycles and track event-free survival, overall survival, and stem cell transplantation rates over up to two years.

A Randomized Study of ASTX727 With or Without Iadademstat in Advanced Myeloproliferative Neoplasms (MPNs)