NCT05210790进行中（未招募）3 期
A Phase 3 Study of the Hepcidin Mimetic Rusfertide (PTG-300) in Patients With Polycythemia Vera
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 293
- 试验地点
- 171
- 主要终点
- Proportion of subjects achieving a response who receive rusfertide compared to placebo.
研究概览
简要总结
The study is designed to evaluate the safety and efficacy of rusfertide in subjects with polycythemia vera (PV) in maintaining hematocrit control and in improving symptoms of PV.
详细描述
Phase 3 study in approximately 250 subjects previously diagnosed with polycythemia vera (PV) who require phlebotomy on a routine basis. There is a 32-week period during which rusfertide or placebo will be added-on to each subject's ongoing therapy for polycythemia vera which may include phlebotomy only or phlebotomy plus stable doses of either of hydroxyurea, interferon and/or ruxolitinib. All subjects who successfully complete the double blind 32-week portion of the study will receive rusfertide for 124 weeks. Approximately 6 and 12 months after their last dose of rusfertide, subjects will have a post-study contact (e.g. by phone) for safety.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All subjects must meet ALL of the following inclusion criteria to be enrolled. There are additional inclusion criteria.
- •Male and female subjects aged 18 (or the country specific minimum age of consent >18) years or older.
- •Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera.
- •At least 3 phlebotomies due to inadequate hematocrit control in 6 months before randomization or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before randomization.
- •CBC values immediately prior to randomization:
- •Hematocrit <45%,
- •WBC 4000/μL to 20,000/μL (inclusive), and
- •Platelets 100,000/μL to 1,000,000/μL (inclusive)
- •Subjects receiving cytoreductive therapy at randomization must be on a stable PV therapy regimen.
- •Subjects treated with phlebotomy alone at randomization must have stopped cytoreductive therapy 2 to 6 months before screening.
排除标准
- •Subjects must meet NONE of the following exclusion criteria to be enrolled. There are additional exclusion criteria.
- •Clinically meaningful laboratory abnormalities at Screening.
- •Subjects who require phlebotomy at hematocrit levels lower than 45%.
- •Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 2 months prior to randomization.
- •Active or chronic bleeding within 2 months prior to randomization.
- •History of invasive malignancies within the last 5 years, except localized cured prostate cancer and cervical cancer.
- •Subjects with in situ or stage 1 squamous cell carcinoma of the skin, in situ or stage 1 basal cell carcinoma of the skin, or in situ melanoma of the skin identified during screen unless the cancer is adequately treated before randomization.
- •Received Busulfan, Pipobroman or 32Phosphorus within 7 months prior to screening.
研究组 & 干预措施
Rusfertide
Experimental
Rusfertide (32 Weeks) - Rusfertide (124 Weeks Open-label)
干预措施: Rusfertide (Drug)
Placebo
Experimental
Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label)
干预措施: Placebo (Drug)
Placebo
Experimental
Placebo (32 Weeks) - Rusfertide (124 Weeks Open-label)
干预措施: Rusfertide (Drug)
结局指标
主要结局
Proportion of subjects achieving a response who receive rusfertide compared to placebo.
时间窗: Week 20 through Week 32
Response is defined as absence of phlebotomy eligibility.
次要结局
- Proportion of subjects with HCT values <45% for rusfertide and placebo.(Week 0 to Week 32)
- Comparison of mean number of phlebotomies between rusfertide and placebo.(Week 0 to Week 32)
- Comparison mean change from baseline in total fatigue score based on PROMIS Short Form between rusfertide and placebo.(Week 32)
- Comparison of mean change from baseline in total MFSAF total score.(Week 32)
研究者
研究点 (171)
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相关资讯
FDA Approves MIMRYLO (rusfertide), First-in-Class Hepcidin Mimetic for Polycythemia Vera- The U.S. FDA approved Takeda's MIMRYLO (rusfertide), a first-in-class hepcidin mimetic, for treating erythrocytosis in adults with polycythemia vera (PV), a blood cancer.
- The approval is supported by the Phase 3 VERIFY trial, in which 76.9% of patients achieved clinical response during Weeks 20-32.
- MIMRYLO demonstrated hematocrit control, reduced phlebotomy burden, and improved fatigue, with injection site reactions and anemia as the most common adverse events.
- The therapy addresses a significant unmet need, as an estimated 78% of PV patients still experience uncontrolled hematocrit with current standard of care.16 days agoRusfertide Achieves Primary Endpoint in Phase 3 VERIFY Trial for Polycythemia Vera- Rusfertide significantly reduced the need for phlebotomies in polycythemia vera (PV) patients, with 77% achieving clinical response compared to 33% on placebo.
- The VERIFY trial met all key secondary endpoints, including hematocrit control and improved patient-reported outcomes, demonstrating comprehensive efficacy.
- The hepcidin mimetic was well-tolerated, with no new safety concerns, suggesting a favorable risk-benefit profile for PV patients.
- Protagonist and Takeda plan to submit detailed results for presentation at upcoming medical conferences and to regulatory agencies for potential approval.last year