A Phase 1, Open-Label, Multi-Center, Safety and Efficacy Study of PRT12396 in Participants With Polycythemia Vera and Myelofibrosis
This is a first-in-human, open-label, multi-center Phase 1 study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of PRT12396 in participants with high-risk polycythemia vera (PV) and myelofibrosis (MF), and to determine the maximum tolerated dose (MTD) and recommended dose(s) for expansion (RDE\[s\]). The study consists of a dose-escalation phase followed by a dose-expansion phase to further evaluate selected dose level(s).
• Willing and able to comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations (including contraception requirements), and other study procedures.
• Confirmed diagnosis of PV or MF according to WHO 2016 or revised ICC/WHO 2022 criteria
• Documented presence of a JAK2 V617 mutation
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
• Estimate life expectancy of ≥12 weeks per investigator assessment.
• Negative serum or urine pregnancy test and agree to use contraception or maintain true abstinence.
• Adequate organ function and bone marrow reserves (hematology, renal, and hepatic)