NCT07469891

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

A Phase 1, Open-Label, Multi-Center, Safety and Efficacy Study of PRT12396 in Participants With Polycythemia Vera and Myelofibrosis

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 100 (estimated)
• Sponsor: Prelude Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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).

Detailed description

This first-in-human, open-label, multi-center Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of PRT12396 in participants with high-risk polycythemia vera (PV) and myelofibrosis (MF). Eligible MF populations include participants with intermediate-1, intermediate-2, or high-risk primary MF, as well as post-polycythemia vera MF or post-essential thrombocythemia MF, with evidence of disease burden based on splenomegaly. The study is conducted in two parts: Part 1 (dose escalation) evaluates escalating oral doses of PRT12396 to evaluate safety and tolerability and to determine the maximum tolerated dose (MTD) and recommended dose(s) for expansion (RDE\[s\]). Part 2 (dose expansion) enrolls additional participants at selected dose level(s) to further characterize the safety, tolerability, pharmacokinetics, and preliminary efficacy of PRT12396 in the PV and MF populations. Approximately up to 100 participants are planned for enrollment across both parts of the study.

Conditions

• Polycythemia Vera (PV)
• Myelofibrosis (MF)
• Myeloproliferative Neoplasms (MPNs)
• Post-Polycythemia Vera Myelofibrosis
• Post-Essential Thrombocythemia Myelofibrosis
• Primary Myelofibrosis (PMF)

Interventions

Type	Name	Description
DRUG	PRT12396	PRT12396 is an investigational oral capsule administered twice daily at the assigned dose level or RDE. Capsules are swallowed whole with water and may be taken one hour before or two hours after meals.

Timeline

• Start date: 2026-04-01
• Primary completion: 2028-04-01
• Completion: 2028-04-01
• First posted: 2026-03-13
• Last updated: 2026-03-31

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study