RecruitingPhase 3NCT07429266

A Study of Sapablursen Evaluating the Safety and Efficacy in Participants With Polycythemia Vera (PV)

A Phase 3 Randomized, Double-blind, Placebo-controlled Global Study of Sapablursen in Polycythemia Vera


Sponsor

Ono Pharmaceutical Co., Ltd.

Enrollment

250 participants

Start Date

Jun 1, 2026

Study Type

INTERVENTIONAL

Conditions

Summary

The purpose of this study is to evaluate the efficacy and safety of sapablursen when added on to current standard of care (SOC) for Polycythemia Vera (PV) therapy. The study will be conducted in three sequential parts (Part 1a blinded treatment, Part 1b open-label treatment, \& Part 2 long-term extension). Participants may receive treatment for up to 156 weeks.


Eligibility

Min Age: 18 Years

Inclusion Criteria5

  • Meet revised 2022 World Health Organization (WHO) and 2022 International Consensus Classification criteria for the diagnosis of PV.
  • Participants must be phlebotomy-dependent.
  • Hct less than (<) 45% at study start.
  • Participants receiving Cytoreduction therapy (CRT) must be on a stable regimen at study start.
  • Adequate organ function and electrolytes.

Exclusion Criteria5

  • Prior treatment of PV with Transmembrane serine protease 6 (TMPRSS6) inhibitors, including sapablursen, or hepcidin mimetics.
  • Clinically significant thrombosis (eg, myocardial infarction, stroke, deep vein thrombosis or splenic vein thrombosis) within 1 month prior to randomization.
  • Participants who require phlebotomy at Hct levels <45%.
  • Meet the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment.
  • Any serious or unstable medical condition or uncontrolled psychiatric condition that would interfere with their ability to comply with study requirements.

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Interventions

DRUGSapablursen

Administered subcutaneously (SC)

DRUGPlacebo

Administered SC


Locations(1)

Regis Clinical Research LLC.

Miami, Florida, United States

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NCT07429266


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