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
试验速览
- 阶段
- 3 期
- 状态
- 撤回
- 发起方
- Sobi, Inc.
- 主要终点
- Cumulative Number of Weeks of Platelet Response
研究概览
简要总结
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.
详细描述
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
- •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.
排除标准
- •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.
研究组 & 干预措施
20 mg Avatrombopag daily
Avatrombopag
干预措施: Avatrombopag 20 mg Oral Tablet (Drug)
结局指标
主要结局
Cumulative Number of Weeks of Platelet Response
时间窗: 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.
次要结局
- Incidence of ITP remission(24 Consecutive Weeks)
- Incidence of subjects achieving a platelet count response(6 Months of Active Treatment)
- Durable Platelet Response(8 Weeks of Treatment)