A Phase 2, Parallel Group, Rollover Study of AKR-501 in Patients With Chronic Idiopathic Thrombocytopenic Purpura (ITP) Who Completed 28 Days of Study Treatment in Protocol 501-CL-003
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Eisai Inc.
- 入组人数
- 53
- 试验地点
- 12
- 主要终点
- Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
The purpose of this study is to determine the safety and efficacy of AKR-501 (avatrombopag) administered in participants with chronic Idiopathic Thrombocytopenic Purpura (ITP) who were enrolled into and completed 28 days of study treatment in Protocol 501-CL-003 (NCT00441090).
详细描述
Participants eligible to enroll into this rollover protocol will begin study treatment within 2-5 days of their Day 28 study termination visit in Protocol 501-CL-003 (NCT00441090). Participants who met the primary efficacy response criterion in Protocol 501-CL-003 will continue receiving the same study treatment to which they were assigned in the previous protocol in a double-blinded manner, these being one of the following 5 treatments:
- avatrombopag 2.5 mg daily
- avatrombopag 5 mg daily
- avatrombopag 10 mg daily
- avatrombopag 20 mg daily
- placebo
Participants who did not meet the primary efficacy response criterion in Protocol 501-CL-003 who otherwise meet the eligibility criteria for this rollover protocol will be offered open label avatrombopag 10 mg daily.
This is a parallel group, rollover study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
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
- •Patients who completed 28 days of study treatment in Protocol 501-CL-
- •No significant safety or tolerability concerns from the patient's participation of Protocol 501-CL-003 as determined by the Investigator.
- •Received medical monitor approval for enrollment into this study.
- •Patients receiving maintenance corticosteroids may be enrolled, as long as the corticosteroids have been administered at a stable dose and the Investigator does not foresee the need to change the steroid dose during study participation. Patients should remain on this stable corticosteroid dose during study participation.
- •Women of child-bearing potential must have a negative serum pregnancy test at the Day 28 assessment in Protocol 501-CL-
- •(Childbearing potential is defined as any woman who has not been surgically sterilized and is pre-menopausal or peri-menopausal i.e., any menstrual flow within 12 months of Screening Visit A for Protocol 501-CL-003).
- •Women of child-bearing potential must agree to practice a medically approved form of contraception (one of the following must be used: condoms (male or female) with a spermicidal agent, diaphragm or cervical cap with a spermicidal agent, IUD,hormonal contraception, abstinence).
- •Willing and able to provide written informed consent.
排除标准
- •Women who are pregnant and/or lactating.
- •Use of the following drugs or treatments:
- •Rituximab
- •Azathioprine, Cyclosporine A, or other immunosuppressant therapy
- •Aspirin, Aspirin-containing compounds, Salicylates,Anticoagulants, Non-steroidal anti-inflammatory drugs(NSAIDs)(including Cyclooxygenase-2 [COX-2] specific NSAIDs), clopidogrel; ticlopidine; and any drugs that affect platelet function.
- •Rh0(D) immune globulin (WinRho®) or intravenous immunoglobulin (IVIG).
- •Inability to comply with protocol requirements or give informed consent, as determined by the Investigator.
- •For more information regarding inclusion/exclusion criteria, please see record for AKR 501-CL-003 Protocol.
研究组 & 干预措施
Placebo (double-blind)
干预措施: Blinded (placebo) (Drug)
Avatrombopag tablets (open-label)
干预措施: Open Label (Avatrombopag tablets) (Drug)
Avatrombopag tablets (double-blind)
干预措施: Blinded (Avatrombopoag tablets) (Drug)
结局指标
主要结局
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
时间窗: Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.
A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of study drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. Related AEs included those whose relationship was categorized as possible or probably by the investigator. Dose interruption includes dose decreased, dose previously stopped, permanently stopped, or temporarily stopped. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag).
Incidence of Severe (Grade 3 or 4) TEAEs
时间窗: Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.
A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag).
Incidence of Drug-Related TEAEs
时间窗: Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.
Drug-related TEAEs included those whose relationship was categorized as possible or probably by the investigator. A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag).
次要结局
- Median Platelet Counts at Selected Analysis Timepoints(Day 1, Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4)
- Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit(Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4)
- Percentage of Participants Who Maintained a Platelet Count of 100,000/mm^3 or Higher by Response Status(Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4)
- Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit(Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4)
- Percentage of Participants Who Maintained Response-Level Platelet Count(Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4)
- Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response(Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4)
- Number of Participants With Changes in Concomitant Steroid Use(Day 1 through last 8 weeks of the Treatment Period)