Phase 1 Study Evaluating Oral PRT12396 for Treating Polycythemia Vera and Myelofibrosis to Assess Safety and Dosage

What this Trial Is About

Researchers are evaluating PRT12396, an investigational oral drug, in participants with high-risk polycythemia vera PV and myelofibrosis MF through a first-in-human, open-label, multi-center Phase 1 study. The study aims to assess the safety, tolerability, pharmacokinetics, and early effects of PRT12396 while determining the maximum tolerated dose and recommended dose for further study. Participants include those diagnosed with PV or MF, including specific subtypes with evidence of disease burden such as splenomegaly. The study is divided into two parts a dose-escalation phase where increasing oral doses of PRT12396 are given twice daily to evaluate safety and identify the best dose levels, followed by a dose-expansion phase where additional participants receive the recommended dose to further assess safety and early efficacy. Capsules are taken twice daily, swallowed whole with water, and may be taken one hour before or two hours after meals. Participants will be involved for an average of about two years, during which researchers will monitor dose-limiting toxicities, adverse events, dose modifications, and pharmacokinetic measures such as blood drug concentration. Other assessments include blood counts, spleen size, symptom scores, and patient global impressions of change. Safety and response outcomes will be tracked throughout the study to guide future research and treatment options.

A Phase 1 Study of PRT12396 in Participants With Select Myeloproliferative Neoplasms