Study of PBSS1113 Combined With Azacitidine for Adults With Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

What this Trial Is About

Researchers are evaluating a new combination treatment for patients with acute myeloid leukemia AML and myelodysplastic syndromes MDS, which are complex blood cancers with limited targeted therapies. The study focuses on patients who have relapsed or refractory AMLMDS or those newly diagnosed but unfit for intensive chemotherapy. The investigational drug PBSS1113, combined with azacitidine, is being studied to assess its safety, how the body processes it, and its preliminary effectiveness in these patient groups. Participants will receive PBSS1113 tablets along with azacitidine in a dose-escalation and cohort expansion trial. This Phase IbII open-label study involves sequential treatment cycles where PBSS1113 is given in combination with azacitidine. The study allows patients who have not received prior intensive chemotherapy or those with relapsed disease to participate. The treatment regimen and dosing schedule will be closely monitored throughout the trial. During the study, participants will undergo regular assessments including laboratory tests to monitor organ function and blood counts. Researchers will evaluate the composite complete remission rate for AML patients and overall response rate for those with MDS after each 28-day treatment cycle. Safety and tolerability will also be regularly assessed. The study will continue until September 2027, with follow-up visits to track patient outcomes and collect pharmacokinetic data.

PBSS1113 in Combination With Azacitidine to Treat Patients With AML/MDS