Polycythemia Vera Symptom Study Evaluating Ruxolitinib Versus Hydroxyurea in a Randomized, Multicenter, Double-Blind, Double-Dummy, Phase 3 Efficacy and Safety Study of Patient Reported Outcomes
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 110
- 主要终点
- Percentage of Subjects Achieving a ≥ 50% Improvement From Baseline in Total Symptom Score-Cytokine (TSS-C) at Week 16, as Measured by the Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Diary
研究概览
简要总结
The purpose of the RELIEF study is to compare symptoms in polycythemia vera (PV) subjects treated with ruxolitinib versus subjects treated with hydroxyurea (HU) as measured by the percent of subjects who achieve a clinically meaningful symptom improvement (ie, total symptom score reduction of ≥ 50% reduction) at Week 16 compared to Baseline. The study is also designed to demonstrate that these responses are durable with continued treatment.
详细描述
This is a Phase 3 multicenter, double-blind, double-dummy, randomized study. Only subjects with PV who have received HU for at least 12 weeks, have been receiving a stable dose before screening, and still have symptoms related to PV will be enrolled.
Subjects will be randomized (1:1) to 1 of 2 treatment arms:
A: ruxolitinib and HU-placebo B: HU and ruxolitinib-placebo
Subjects randomized to either arm may be eligible to transition to open-label ruxolitinib after Week 16.
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
- Quadruple (Participant, Care Provider, 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
- •Subjects must currently be reporting symptoms while on a stable dose of HU monotherapy and be eligible to continue HU on study after randomization.
- •Before screening, the subject must have been receiving HU for at least 12 weeks AND be receiving a stable dose.
- •Subjects must meet baseline symptom criteria
- •Subjects should meet at least 1 of the following criteria:
- •No more than 2 phlebotomies within the 6 months before screening OR
- •No palpable splenomegaly.
- •Subjects must have a hematocrit that can be controlled within 35% to 48% (inclusive) before randomization.
排除标准
- •Subjects with inadequate liver or renal function at screening.
- •Subjects with clinically significant infection that requires therapy
- •Subjects with known active hepatitis A, B, or C at screening or with known HIV positivity.
- •Subjects with an active malignancy over the previous 2 years
- •Subjects with clinically significant cardiac disease (Class III or IV).
研究组 & 干预措施
ruxolitinib and hydroxyurea (HU)-placebo
干预措施: Ruxolitinib (Drug)
ruxolitinib and hydroxyurea (HU)-placebo
干预措施: HU-placebo (Drug)
HU and ruxolitinib-placebo
干预措施: Hydroxyurea (HU) (Drug)
HU and ruxolitinib-placebo
干预措施: Ruxolitinib-placebo (Drug)
结局指标
主要结局
Percentage of Subjects Achieving a ≥ 50% Improvement From Baseline in Total Symptom Score-Cytokine (TSS-C) at Week 16, as Measured by the Modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Diary
时间窗: From Baseline to Week 16
Symptoms of polycythemia vera were assessed using a modified Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) electronic diary. Using the diary, patients rated the following symptoms on a scale from 0 (absent) to 10 (worst imaginable): tiredness, itching, muscle aches, night sweats, and sweats while awake. The total symptom score ranged from 0-50 and was calculated as the sum of the 5 symptom scores. A higher score indicates worse symptoms.
次要结局
- Percentage of Subjects Achieving ≥ 50% Improvement From Baseline in the Individual Symptom Scores for TSS-C at Week 16(From Baseline to Week 16)
- Proportion of Subjects Randomized to Ruxolitinib Who Achieved ≥ 50% Improvement From Baseline in Total Symptom Score-Cytokine and the Individual Symptom Scores at Week 16 That Were Maintained at Week 48(Week 48)