RecruitingInterventionalPhase 3

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

NCT ID: NCT07429266Sponsor: Ono Pharmaceutical Co., Ltd.Last updated: 2026-06-05

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.

Arms & interventions

DrugSapablursen
Administered subcutaneously (SC)
DrugPlacebo
Administered SC

Outcome measures

Primary

Percentage of Participants with Absence of Phlebotomy Eligibility
Response is defined as absence of phlebotomy eligibility.
Time frame: Week 20 through Week 32

Secondary

Number of Phlebotomies
Time frame: Week 0 through Week 32
Percentage of Participants with Hct Control
Time frame: Week 0 to Week 32
Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form Total T-score
Time frame: Baseline, Week 32
Change from Baseline in Myelofibrosis Symptom Assessment Form (MFSAF) Total Symptom Score (TSS)
Time frame: Baseline, Week 32
Percentage of Participants with Absence of Phlebotomy Eligibility
Time frame: Week 20 through Week 52
Number of Phlebotomies
Time frame: Week 0 through Week 52
Percentage of Participants with Hct Control
Time frame: Week 0 through Week 52

Eligibility criteria

Sex: AllAge: 18 Years and olderHealthy volunteers: No

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.

Study locations (1)

Regis Clinical Research LLC.

Miami, Florida, 33126

Recruiting

Roberto Reyes · Contact

305-546-3952 rreyes@regisclinical.com

Yilena Rodriguez Marrero, MD · Principal Investigator