2025-524669-25-00招募中3 期
A Phase 3 Randomized, Double-blind, Placebo-controlled Global Study of Sapablursen in Polycythemia Vera
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 106
- 试验地点
- 76
- 主要终点
- Number of phlebotomies from Week 0 through Week 32 Hct control, defined as all local laboratory Hct <45% from Week 0 through Week 32 without phlebotomy. A single Hct value ≥45% is allowed. Hct values from each clinical site’s local laboratory will be used for this endpoint
研究概览
简要总结
To compare the clinical efficacy of sapablursen vs placebo in participants with PV through 32 weeks
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •1 Written informed consent by the participant prior to Screening, participant who understands the study procedures and is able to adhere to study requirements
- •2 Meets revised 2022 World Health Organisation (WHO) and 2022 International Consensus Classification (ICC) criteria for the diagnosis of PV
- •3 Participants must be phlebotomy-dependent
- •4 Hct less than (<) 45% at study start
- •5 Adequate organ function and electrolytes
- •6 Participants receiving Cytoreduction therapy (CRT) must be on a stable regimen at study start.
排除标准
- •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 less than (<) 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 as judged by the Investigator that would interfere with their ability to comply with study requirements.
- •6 Female participants who are pregnant, planning to become pregnant during the study, or breastfeeding.
研究组 & 干预措施
Sapablursen, Sapablursen
Test
干预措施: Sapablursen (Drug)
Matching placebo to Sapablursen
Placebo
干预措施: Matching placebo to Sapablursen (Drug)
结局指标
主要结局
Number of phlebotomies from Week 0 through Week 32 Hct control, defined as all local laboratory Hct <45% from Week 0 through Week 32 without phlebotomy. A single Hct value ≥45% is allowed. Hct values from each clinical site’s local laboratory will be used for this endpoint
Number of phlebotomies from Week 0 through Week 32 Hct control, defined as all local laboratory Hct <45% from Week 0 through Week 32 without phlebotomy. A single Hct value ≥45% is allowed. Hct values from each clinical site’s local laboratory will be used for this endpoint
次要结局
- Key secondary: Response, defined as absence of phlebotomy eligibility, starting from Week 20 through Week 32. Phlebotomy eligibility is defined as either of the following: - Hct ≥45% and at least 3% (absolute) higher than the baseline Hct confirmed through 2 consecutive assessments OR Hct ≥48%. Hct values from each clinical site’s local laboratory will be used for the primary endpoint to allow same day phlebotomy decisions.
- Key secondary: Change from baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS Fatigue SF-8a) total T-score (8 items) at Week 32; • Change from baseline in the Myelofibrosis Symptom Assessment Form version 4.0 Total Symptom Score (MFSAF v4.0 TSS) (7 items) at Week 32
- Other Secondary: For participants originally randomized to sapablursen: • Response, defined as absence of phlebotomy eligibility, from Week 20 through Week 52; • Number of phlebotomies from Week 0 through Week 52; • Hct control from Week 0 through Week 52
- Safety: • Frequency of treatment-emergent adverse events (TEAEs); • Frequency of serious adverse events • Frequency of TEAEs leading to dose reduction, interruption, or discontinuation of study treatment
研究者
Clinical Trial Information
Scientific
Deciphera Pharmaceuticals Inc.
研究点 (76)
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