Randomized, Open Label, Multicenter Phase III Study of Efficacy and Safety in Polycythemia Vera Subjects Who Are Resistant to or Intolerant of Hydroxyurea: JAK Inhibitor INC424 Tablets Versus Best Available Care (The RESPONSE Trial)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 222
- 主要终点
- The Percentage of Participants Achieving a Primary Response at Week 32
研究概览
简要总结
This pivotal phase III trial (CINC424B2301) is designed to compare the efficacy and safety of ruxolitinib (INC424) to Best Available Therapy (BAT) in participants with polycythemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU).
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
- None
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
- •Participants diagnosed with PV for at least 24 weeks prior to screening according to the 2008 World Health Organization criteria
- •Participants resistant to or intolerant of hydroxyurea
- •Participants with a phlebotomy requirement
- •Participants with splenomegaly (palpable or non-palpable) and a spleen volume, as measured by MRI (or CT in applicable participants ), of greater than or equal to 450 cubic centimeters
- •Participants with an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
排除标准
- •Women who are pregnant or nursing
- •Participants with inadequate liver or renal function
- •Participants with significant bacterial, fungal, parasitic, or viral infection requiring treatment
- •Participants with an active malignancy within the past 5 years, excluding specific skin cancers
- •Participants with known active hepatitis or HIV positivity
- •Participants who have previously received treatment with a JAK inhibitor
- •Participants being treated with any investigational agent
研究组 & 干预措施
ruxolitinib tablets
Starting dose of 10 mg BID with individualized dose titration ranging from 5 mg once a day (QD) to 25 mg BID based on safety and efficacy
干预措施: ruxolitinib tablets (Drug)
Best Available Therapy
Best Available Therapy (BAT) will be selected by the Investigator for each participant. BAT may not include experimental agents (i.e. those not approved for the treatment of any indication) as well as a limited number of other selected drugs in accordance with the protocol-defined requirements.
干预措施: Best Available Therapy (BAT) (Other)
结局指标
主要结局
The Percentage of Participants Achieving a Primary Response at Week 32
时间窗: 32 Weeks
Primary response was defined as having achieved hematocrit control (the absence of phlebotomy eligibility beginning at the Week 8 visit and continuing through Week 32) and Spleen Volume Reduction (a greater than or equal to 35% reduction from baseline in spleen volume at Week 32).
次要结局
- The Percentage of Participants Achieving Complete Hematological Remission at Week 32(32 Weeks)
- The Percentage of Participants Who Achieved Durable Spleen Volume Reduction at Week 48(48 Weeks)
- The Percentage of Participants Achieving a Durable Complete or Partial Clinicohematologic Response at Week 48(48 Weeks)
- Duration of the Absence of Phlebotomy Eligibility(256 Weeks)
- The Percentage of Participants Achieving a Durable Primary Response at Week 48(48 Weeks)
- The Percentage of Participants Who Achieved a Durable Hematocrit Control at Week 48(48 Weeks)
- Duration of Reduction in Spleen Volume(256 Weeks)
- The Percentage of Participants Who Achieved a Durable Complete Hematological Remission at Week 48(48 Weeks)
- Estimated Duration of the Primary Response(Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study)
- The Percentage of Participants Who Achieved Overall Clinicohematologic Response at Week 32(32 Weeks)
- Estimated Duration of the Complete Hematological Remission(Through study completion, analysis was conducted when all participants had completed the Week 80 visit or discontinued the study)
- Duration of The Overall Clinicohematologic Response(256 Weeks)