A Phase 2 Interventional Single Arm Study Describing Platelet Responses and ITP Remission Rates in Adult Subjects With Immune Thrombocytopenia Purpura Receiving Romiplostim
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Amgen
- 入组人数
- 75
- 试验地点
- 48
- 主要终点
- Number of Months With Platelet Response During the 12-Month Treatment Period
研究概览
简要总结
The purpose of this study is to describe the number of months with a platelet response over a 12 month treatment period and to describe ITP remission rates in adults with ITP receiving romiplostim.
详细描述
The study includes a 4-week screening period, a 12-month romiplostim treatment period, and a romiplostim dose-tapering period.
During the 12-month treatment period romiplostim doses could be increased or decreased to maintain a platelet count between ≥ 50 x 10^9/L and ≤ 200 x 10^9/L. Participants who dose reduce such that they no longer require treatment with romiplostim during the 12-month treatment period will continue with all required study procedures up to 12 months and will be monitored for ITP remission for at least 6 months.
At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10^9/L will enter the tapering period, during which the romiplostim dose will be decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. If a participant maintains a platelet count of ≥ 50 x 10^9/L in the absence of romiplostim and all medications for ITP (concomitant or rescue), the participant will be followed for at least 6 months to confirm the incidence of ITP remission. If a participant's platelet count falls below 50 x 10^9/L and the participant has tapered off treatment with romiplostim, the participant will enter the stabilization period and reinitiate romiplostim for up to 8 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 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 has been diagnosed with primary ITP according to the American Society of Hematology (ASH) guidelines (George et al, 1996) and previously received only 1st line therapies.
- •First line therapy is defined as corticosteroids, immunoglobulin G (IVIG), anti-D and vinca alkaloids (used for the treatment of ITP related thrombocytopenia only). A platelet transfusion at any time during the six month period since the original diagnosis would not exclude the subject from study participation
- •Initial diagnosis of primary ITP within 6 months of enrollment
- •Age ≥ 18 years at screening
- •A single platelet count ≤ 30 x 10⁹/L at any time during the screening period
- •Subject or subject's legally acceptable representative has provided informed consent
排除标准
- •Known history of a bone marrow stem cell disorder
- •Surgical resection of the spleen
- •Subject has a history of cancer or current malignancy other than basal cell carcinoma or cervical cancer in-situ with active treatment or disease within 5 years of screening
- •Known history of congenital thrombocytopenia
- •Known history of hepatitis B, hepatitis C, or human immunodeficiency virus
- •Positive H. pylori by urea breath test or stool antigen test at screening
- •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 recurrent venous thromboembolism or thrombotic events or an occurrence within 5 years of enrollment.
- •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 mercaptopurine (6-MP) or anticipated use during the time of the proposed study
- •All hematopoietic growth factors including interleukin-11 (IL-11) (oprelvekin) within 4 weeks before the screening visit
- •Alkylating agents use at any time before or during the screening visit or anticipated during the time of the proposed study
- •Known hypersensitivity to any recombinant E. coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune)
- •Currently enrolled in another investigational device or drug study, or less than 30 days since ending another investigational device or drug study(s), or receiving other investigational agent(s)
- •Subject will have any other investigational procedures performed while enrolled in this clinical study
- •Subject is pregnant or breast feeding, or planning to become pregnant within 5 weeks after the end of treatment
- •Female subject of child bearing potential is not willing to use, in combination with her partner, highly effective contraception during treatment and for 4 weeks after the end of treatment
- •Subject has previously enrolled into a romiplostim study
- •Subject will not be available for protocol required study visits, to the best of the subject's and investigator's knowledge
- •Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures
研究组 & 干预措施
Romiplostim
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with 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 (Biological)
结局指标
主要结局
Number of Months With Platelet Response During the 12-Month Treatment Period
时间窗: 12 months
The primary endpoint was the number of months a participant achieved a platelet response during the 12-month treatment period. A platelet response for any 1 month was defined as the median of platelet counts measured in the month ≥ 50 x 10\^9/L. Platelet counts within 4 weeks following a rescue medication use or following splenectomy were considered non-response. Months without any platelet count measurement were considered as months with no platelet response.
次要结局
- Percentage of Participants With Splenectomy During the 12-month Treatment Period(12 months)
- Number of Participants Who Developed Antibodies to Romiplostim(Baseline and at end of treatment (based on response to treatment, this could occur between 12 months and approximately 18 months))
- Percentage of Participants With ITP Remission(Up to 24 months)
- Number of Participants With Adverse Events(From first dose date of romiplostim to end of study (up to 24 months).)