NCT01444417已完成3 期
A Phase 3 Randomized, Double Blind, Placebo Controlled Study to Determine the Safety and Efficacy of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 62
- 试验地点
- 1
- 主要终点
- Percentage of Participants With a Durable Platelet Response
研究概览
简要总结
The purpose of this study is to evaluate the efficacy of romiplostim in the treatment of thrombocytopenia in pediatric patients with Immune thrombocytopenia purpura (ITP) as measured by durable platelet response.
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
- 1 Year 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of primary ITP according to the American Society of Hematology (ASH) guidelines at least 6 months prior to screening, regardless of splenectomy status
- •Subject must be refractory to a prior ITP therapy, having relapsed after at least 1 prior ITP therapy, or ineligible for other ITP therapies; prior therapy includes first-line therapies
- •Age ≥ 1 year and < 18 years at the time of providing informed consent
- •The mean of 2 platelet counts taken during the screening period must be ≤ 30 x 10^9/L with neither count > 35 x 10^9/L
- •A serum creatinine concentration ≤ 1.5 times the laboratory normal range (for each age category) during the screening period
- •Adequate liver function; serum bilirubin ≤ 1.5 times the laboratory normal range during the screening period; aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3.0 times the laboratory normal range during the screening period
- •Hemoglobin > 10.0 g/dL during the screening period
- •Subject and/or subject's legally acceptable representative has provided informed consent prior to any study-specific procedure; subject has provided assent, where required
排除标准
- •Known history of a bone marrow stem cell disorder; any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled in the study
- •Known active or prior malignancy except adequately treated basal cell carcinoma
- •Known history of congenital thrombocytopenia
- •Known history of hepatitis B, hepatitis C, or human immunodeficiency virus (HIV)
- •Known history of H. pylori by urea breath test or stool antigen test within 6 months of enrollment or successfully treated with no evidence of infection
- •Known history of systemic lupus erythematosus, evans syndrome, or autoimmune neutropenia
- •Known history of antiphospholipid antibody syndrome or positive for lupus anticoagulant
- •Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura
- •Previous history of venous thromboembolism or thrombotic events
- •Previous use of romiplostim, pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), Eltrombopag, recombinant human thrombopoietin (rHuTPO) or any platelet producing agent
- •Rituximab (for any indication) or 6-mercaptopurine (6-MP) within 14 weeks before the screening visit, or anticipated use during the time of the proposed study
- •Splenectomy within 4 weeks of the screening visit
- •All hematopoietic growth factors including interleukin-11 (IL-11) (oprelvekin) within 4 weeks before the screening visit
- •Alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
- •Vaccinations known to decrease platelet counts within 8 weeks before the screening visit
- •Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
- •Other criteria may apply
研究组 & 干预措施
Romiplostim
Experimental
Participants received once weekly subcutaneous romiplostim for 24 weeks at a starting dose of 1 µg/kg; weekly dose increases continued in increments of 1 µg/kg/week to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L.
干预措施: Romiplostim (Drug)
Placebo
Placebo Comparator
Participants received weekly subcutaneous placebo for 24 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage of Participants With a Durable Platelet Response
时间窗: Week 18 to week 25
A participant with durable platelet response was defined as achieving at least 6 weekly platelet counts of ≥ 50 x 10\^9/L during the last 8 weeks of treatment (platelet counts obtained from week 18 to week 25). If a platelet count from a participant was not available (missing) in a certain week, that week was imputed as non-response for that participant. Platelet counts were not deemed as a positive response for 4 weeks after the administration of rescue medication.
次要结局
- Percentage of Participants With an Overall Platelet Response(Week 2 to week 25)
- Number of Weeks With Platelet Response(Week 2 to week 25)
- Percentage of Participants Who Received Rescue Medication During the Treatment Period(24 weeks)
- Total Number of Composite Bleeding Episodes(Week 2 to week 25)
- Number of Participants With Adverse Events(From the first dose of study drug until 4 weeks after last dose; 28 weeks.)
研究者
研究点 (1)
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