A Phase 3, Randomized, Multicenter, Open-label Study to Evaluate the Efficacy and Safety of Romiplostim Plus Predniso(lo)ne vs. Predniso(lo)ne Alone for the Treatment of Adults With Previously Untreated Primary Immune Thrombocytopenia (ITP).
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Amgen
- 入组人数
- 126
- 试验地点
- 3
- 主要终点
- Percentage of Participants With a Durable Platelet Response (DPR)
研究概览
简要总结
This Phase 3 study is designed to evaluate the efficacy and safety of romiplostim in combination with predniso(lo)ne compared with predniso(lo)ne alone in adults with previously untreated Primary Immune Thrombocytopenia (ITP).
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 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥18 years or adult legal age within country if older than 18 years.
- •Diagnosis of primary ITP according to the 2019 International Consensus (ICR) that is previously untreated and requires treatment.
- •Note: The investigator should ensure that the diagnosis of primary ITP is established by excluding other causes of isolated thrombocytopenia, as outlined in the 2019 ICR, which states that the diagnosis of primary ITP is principally based on the exclusion of other causes of isolated thrombocytopenia.
- •Note: If emergency treatment is necessary, platelet count performed before emergency can be used for study inclusion.
- •Note: Emergency ITP treatment with any thrombopoietin receptor agonists (TPO-RAs), or splenectomy is not allowed.
- •Platelet count < 30 × 10^9/L or Platelet count < 50× 10^9/L with clinically significant bleeding before any medical intervention.
排除标准
- •Life-threatening bleeding at randomization.
- •Known sensitivity or intolerance to any of the products to be administered during study (eg, uncontrolled diabetes) or to any Escherichia coli-derived product (eg, filgrastim, pegfilgrastim, certain insulins).
- •Uncontrolled hypertension before randomization.
- •Abnormal hepatic or renal function at screening.
- •History of total splenectomy.
- •Use of concurrent anticoagulation therapy and/or antiplatelet therapy.
- •Need for nonsteroidal anti-inflammatory drugs (NSAIDs) use and use of NSAIDs within 7 days before randomization.
- •Venous or arterial thrombotic event within 3 or 6 months, respectively, before randomization.
- •Other protocol-defined Inclusion/Exclusion may apply.
研究组 & 干预措施
Romiplostim + Predniso(lo)ne
Participants will receive romiplostim administered subcutaneously (SC) in combination with predniso(lo)ne administered orally during Part 1 of the study.
Participants who complete Part 1 of the study will enter Part 2 and continue participation for study assessments.
干预措施: Predniso(lo)ne (Drug)
Predniso(lo)ne
Participants will receive predniso(lo)ne administered orally during Part 1 of the study.
Participants who complete Part 1 of the study will enter Part 2 and continue participation for study assessments.
干预措施: Predniso(lo)ne (Drug)
Romiplostim + Predniso(lo)ne
Participants will receive romiplostim administered subcutaneously (SC) in combination with predniso(lo)ne administered orally during Part 1 of the study.
Participants who complete Part 1 of the study will enter Part 2 and continue participation for study assessments.
干预措施: Romiplostim (Drug)
结局指标
主要结局
Percentage of Participants With a Durable Platelet Response (DPR)
时间窗: 8 weeks
次要结局
- Time to Next Treatment (TTNT)(12 months)
- Cumulative Exposure to Corticosteroids(12 months)
- Change From Baseline in ITP Patient Assessment Questionnaire (ITP-PAQ)(12 months)
- Change in Summary Scores and Visual Analogue Scale (VAS) Scores per EuroQol 5-Dimension 5-Level (EQ 5D-5L)(12 months)
- Incidence of Hospitalization and Rescue Medication in Part 1(6 months)
- Number of Participants Experiencing Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Treatment-Emergent Adverse Events of Interest (EOI), and Fatal TEAEs(12 months)
- Percentage of Participants With Clinically Significant Bleeding Events in the Immune Thrombocytopenia-specific Bleeding Assessment Tool (ITP-BAT)(6 months)
- Serum Trough Concentration (Ctrough) of Romiplostim(6 months)
- Number of Participants With Anti-Romiplostim Antibodies and Anti-Thrombopoietin (TPO) Antibodies(6 months)