Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With ITP of ≤6 Months
2021年10月15日 更新者:Sobi, Inc.
A Multicenter, Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With Immune Thrombocytopenia (ITP) of ≤6 Months Duration
This study will evaluate the safety and efficacy of avatrombopag in subjects with a confirmed diagnosis of primary ITP (≤6 months duration) over 26 weeks of treatment, and also evaluate the incidence of ITP remission.
研究概览
详细说明
This phase 3b, multi-center, open-label study will enroll approximately 75 adult subjects with a confirmed diagnoses of primary ITP (≤6 months duration) who have had a previous response to a first line treatment.
The study will consist of a 26-week treatment period to evaluate the safety and efficacy of avatrombopag.
Subjects with platelet counts ≥50×10⁹/L at Week 26 may enter a dose-tapering period in which the dose of avatrombopag will be decreased for up to 16 weeks until avatrombopag treatment is discontinued and the platelet count is maintained ≥50×10⁹/L.
Once avatrombopag treatment has been discontinued, the subjects will enter a remission evaluation period of up to 24 weeks to evaluate whether they have entered a state of remission.
研究类型
介入性
阶段
- 第三阶段
参与标准
研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
18年 及以上 (成人、年长者)
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion Criteria:
- Male or female subjects ≥18 years of age at Screening.
- Subject must be able to provide informed consent.
- Subject has a confirmed diagnosis of primary ITP according to the International Consensus Report on the Investigation and Management of Primary ITP within the previous 6 months prior to Visit 1 and has had a previous response to a first line treatment (corticosteroids, IVIg, or anti-D), in the opinion of the Investigator.
- Subject has at least one platelet count <30×10⁹/L at any time during the screening period or at the Baseline visit.
- Females of childbearing potential must have a negative pregnancy test at Screening and Baseline.
- Female subjects of childbearing potential who are sexually active and male subjects who are sexually active must agree to use effective methods of contraception.
- Subject is willing and able to comply with all aspects of the protocol.
Exclusion Criteria:
- Thrombocytopenia due to a known condition other than primary ITP (e.g., systemic lupus erythematosus, H. pylori infection, splenomegaly, chronic liver disease).
- Any history of arterial or venous thrombosis, including partial or complete thrombosis (history of superficial thrombophlebitis is not exclusionary).
- Subjects with known inherited thrombocytopenia (e.g., MYH-9 disorders).
- History of myelodysplastic syndrome (MDS) or other hematologic malignancies.
- Current history of significant cardiac arrhythmias or decompensated congestive heart failure.
- History of hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV).
- Previous use of eltrombopag, romiplostim, recombinant human TPO or other platelet-producing agents.
- Surgical resection of the spleen.
- Previous use of mycophenolate mofetil (MMF), rituximab (or other B-cell lymphocyte depleting agents), mercaptopurine (6-MP) or alkylating agents.
- Concurrent malignant disease, other than non-melanoma skin cancer or cervical cancer in-situ.
- Known allergy to avatrombopag or any of its excipients.
- Subject is unable to take oral medication or has a malabsorption syndrome or any other uncontrolled gastrointestinal condition.
- Enrollment in another clinical study with any investigational drug or device within 30 days of Day 1/Visit 2 (or 5 half-lives, whichever is longer); however, participation in observational studies is permitted.
- Any clinically relevant abnormality which makes the subject unsuitable for participation in the study, in the opinion of the Investigator.
- Considered unable or unwilling to comply with the study protocol requirements.
学习计划
本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的:20 mg Avatrombopag daily
Avatrombopag
|
Avatrombopag administered at a frequency to maintain a target platelet count between ≥50 and ≤150×10⁹/L
其他名称:
|
研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Cumulative Number of Weeks of Platelet Response
大体时间:6 Months of Active Treatment
|
Cumulative number of weeks of platelet response in which the platelet count is ≥50×10⁹/L during 6 months of treatment in the absence of rescue therapy.
|
6 Months of Active Treatment
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Durable Platelet Response
大体时间:8 Weeks of Treatment
|
Durable platelet response as defined by the incidence of subjects who have at least 6 out of 8 weekly platelet counts ≥50×10⁹/L during the last 8 weeks of treatment.
|
8 Weeks of Treatment
|
|
Incidence of ITP remission
大体时间:24 Consecutive Weeks
|
Incidence of ITP remission as defined by platelet count ≥50×10⁹/L for 24 consecutive weeks with no ITP treatments (concomitant or rescue).
|
24 Consecutive Weeks
|
|
Incidence of subjects achieving a platelet count response
大体时间:6 Months of Active Treatment
|
Incidence of subjects achieving a platelet count response (≥50×10⁹/L) during the active treatment period of the study.
|
6 Months of Active Treatment
|
合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
赞助
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (预期的)
2021年10月14日
初级完成 (预期的)
2024年4月6日
研究完成 (预期的)
2025年2月19日
研究注册日期
首次提交
2021年9月8日
首先提交符合 QC 标准的
2021年9月8日
首次发布 (实际的)
2021年9月16日
研究记录更新
最后更新发布 (实际的)
2021年10月22日
上次提交的符合 QC 标准的更新
2021年10月15日
最后验证
2021年10月1日
更多信息
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