A Randomized, Controlled, Open-label Study Evaluating the Efficacy and Tolerability of AMG 531 Versus Medical Standard of Care as Chronic Therapy for Non-splenectomized Subjects With Immune (Idiopathic) Thrombocytopenic Purpura
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 234
- 主要终点
- Number of Participants With Splenectomy During 52-Week Treatment Period
研究概览
简要总结
This is a phase 3b, multi-center, randomized, Standard of Care (SOC)-controlled, open-label, 52-week treatment study to compare romiplostim to medical SOC for Idiopathic Thrombocytopenia Purpura (ITP), with a 6-month Safety Follow-up. Patients randomized to romiplostim must complete the taper or discontinuation of medical SOC for ITP as soon as medically feasible after the initiation of romiplostim. After the completion or discontinuation of the study treatment period, any participant who does not transfer in to another romiplostim study will complete a 6-month Safety Follow-up period.
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
- •Subject is ≥ 18 years of age
- •Subject has a diagnosis of Idiopathic Thrombocytopenia Purpura (ITP) according to the American Society of Hematology (ASH) guidelines
- •If subject is > 60 years of age, subject has a written bone marrow biopsy report confirming the diagnosis of ITP
- •Subject has received at least 1 prior therapy for ITP
- •Subject has a platelet count < 50,000 or their platelet count falls to < 50,000 during or after a clinically-indicated taper or discontinuation of current ITP therapy
- •Before any study-specific procedure, the appropriate written informed consent must be obtained
排除标准
- •Subject has had a splenectomy for any reason
- •Subject has an active malignancy
- •Subject has a history of cancer, other than basal cell carcinoma or cervical carcinoma in situ, with treatment or active disease within 5 years
- •Subject has a known history of bone marrow stem cell disorder
- •Subject has participated in any study evaluating polyethylene glycol-conjugated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), AMG 531, or a thrombopoietic protein
- •Subject is receiving other investigational agents or procedures
- •Subject is currently enrolled in, or has completed within the last 30 days, another investigational device or drug study
- •Subject is pregnant or breast feeding
- •Subject is not using adequate contraceptive precautions
- •Subject has known sensitivity to any recombinant E. coli-derived product
- •Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and does not have a legally acceptable representative
- •Subject has any kind of disorder that compromises the ability of the subject to comply with study procedures
研究组 & 干预措施
Romiplostim
Romiplostim administered by subcutaneous injection once weekly at a starting dose of 3 μg/kg, adjusted to a maximum dose of 10 μg/kg to maintain a platelet count between 50 and 200 x 10^9/L for up to 52 weeks.
干预措施: Romiplostim (Biological)
Standard of Care
Medical standard of care treatments were selected and prescribed by the investigator according to standard institutional practices or therapeutic guidelines and administered for up to 52 weeks.
干预措施: Medical Standard of Care for ITP (Drug)
结局指标
主要结局
Number of Participants With Splenectomy During 52-Week Treatment Period
时间窗: 52 weeks
Occurrence of a splenectomy. Participants who discontinued study during the treatment period prior to reporting a splenectomy were considered as having had a splenectomy.
Number of Participants With Treatment Failure During 52-Week Treatment Period
时间窗: 52 weeks
Treatment failure was defined by platelet counts ≤ 20 x 10\^9/L for 4 consecutive weeks at the highest recommended dose and schedule, a major bleeding event, or change in therapy due to an intolerable side effect or bleeding symptom.
次要结局
- Time to Splenectomy(52 weeks)
- Percentage of Participants With Platelet Response(Weeks 1-8, and Weeks 12, 16, 20, 24, 28, 32, 36, 40, 44, 48 and 52)
- Change in ITP-PAQ Physical Health Domain of Symptoms(Baseline and 52 weeks)
- Change in ITP-PAQ Physical Health Domain of Fatigue(Baseline and 52 weeks)
- Change in ITP-PAQ Physical Health Domain of Bother(Baseline and 52 weeks)
- Change in ITP-PAQ Physical Health Domain of Activity(Baseline and 52 weeks)