Sapablursen added to standard care for people with polycythemia vera

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

This trial will test whether adding sapablursen to standard therapy can reduce the need for phlebotomy in people with polycythemia vera who remain phlebotomy-dependent.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 250 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Ono Pharmaceutical Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Miami, Florida)
• Trial ID: NCT07429266 on ClinicalTrials.gov

What this trial studies

Sapablursen is being tested as an add-on to current standard of care in adults with phlebotomy-dependent polycythemia vera. The phase 3 program is organized in three sequential parts: a blinded treatment period (Part 1a) comparing sapablursen to placebo, an open-label treatment period (Part 1b), and a long-term extension (Part 2) with up to 156 weeks of exposure. Eligible participants must meet the 2022 WHO and ICC diagnostic criteria for PV, be phlebotomy-dependent with baseline hematocrit <45%, and may remain on a stable cytoreductive regimen. Outcomes focus on hematocrit control, need for phlebotomy, and safety monitoring including thrombotic events and other adverse effects.

Who should consider this trial

Good fit: Ideal candidates are adults with PV who meet 2022 WHO/ICC diagnostic criteria, are phlebotomy-dependent with Hct <45% at baseline, and are on stable cytoreductive therapy if applicable.

Not a fit: Patients with recent clinically significant thrombosis, progression to post‑PV myelofibrosis, prior exposure to TMPRSS6 inhibitors or hepcidin mimetics, unstable medical or psychiatric conditions, or those who are not phlebotomy-dependent are unlikely to benefit from this treatment.

Why it matters

Potential benefit: If successful, sapablursen could reduce the frequency of phlebotomy and improve hematocrit control for people with PV.

How similar studies have performed: Hepcidin-modulating approaches and early-phase TMPRSS6 inhibitor studies have shown promising hematologic effects, but robust phase 3 evidence is still limited.

Eligibility criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Where this trial is running

Miami, Florida

• Regis Clinical Research LLC. — Miami, Florida, United States (Recruiting)

Study contacts

• Study coordinator: Clinical Team

Conditions Polycythemia Vera