NCT07429266RecruitingPhase 3
A Phase 3 Randomized, Double-blind, Placebo-controlled Global Study of Sapablursen in Polycythemia Vera
Ono Pharmaceutical Co., Ltd.10 sites in 3 countries250 target enrollmentStarted: May 25, 2026Last updated:
Conditions
Interventions
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Enrollment
- 250
- Locations
- 10
- Primary Endpoint
- Percentage of Participants with Absence of Phlebotomy Eligibility
Study Overview
Brief 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.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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 Criteria
- •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.
Arms & Interventions
Placebo
Experimental
Placebo (32 Weeks) - Sapablursen (124 Weeks Open-label)
Intervention: Sapablursen (Drug)
Placebo
Experimental
Placebo (32 Weeks) - Sapablursen (124 Weeks Open-label)
Intervention: Placebo (Drug)
Sapablursen
Experimental
Sapablursen (32 Weeks) - Sapablursen (124 Weeks Open-label)
Intervention: Sapablursen (Drug)
Outcomes
Primary Outcomes
Percentage of Participants with Absence of Phlebotomy Eligibility
Time Frame: Week 20 through Week 32
Response is defined as absence of phlebotomy eligibility.
Secondary Outcomes
- Percentage of Participants with Hct Control(Week 0 to Week 32)
- Number of Phlebotomies(Week 0 through Week 32)
- Change from Baseline in Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form Total T-score(Baseline, Week 32)
- Change from Baseline in Myelofibrosis Symptom Assessment Form (MFSAF) Total Symptom Score (TSS)(Baseline, Week 32)
- Percentage of Participants with Absence of Phlebotomy Eligibility(Week 20 through Week 52)
- Number of Phlebotomies(Week 0 through Week 52)
- Percentage of Participants with Hct Control(Week 0 through Week 52)
Investigators
Study Sites (10)
Loading locations...
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