NCT05345197进行中（未招募）2 期
Emicizumab in Patients With Acquired Hemophilia A: Multicenter, Single-arm, Open-label Clinical Trial
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 51
- 试验地点
- 16
- 主要终点
- Primary Outcome Meassure
研究概览
简要总结
This is a phase II multicenter open-label, single-arm prospective study to evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
详细描述
Patients with AHA who are eligible will receive two loading doses of the study drug, emicizumab (6mg/kg on day 1 and 3 mg/kg on day 2) followed by once weekly subcutaneous emicizumab (1.5 mg/kg). Immunosuppression will be given concurrently as per investigator discretion. The primary endpoint (bleed rate) will be assessed after 12 weeks on study drug. If partial remission of the AHA has not been achieved, an additional 12 weeks of study drug may be given. A historical cohort and a study conducted in parallel in Germany (NCT04188639) will serve as control groups for evaluation of secondary endpoints provided the study cohort are comparable.
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
- •Signed Informed Consent/Assent Form
- •Age ≥18 years at time of signing Informed Consent Form
- •Ability to comply with the study protocol, in the investigator's judgment
- •Diagnosis of AHA based on a reduced FVIII activity (<50 %) and positive FVIII inhibitor (>0.6 BU/ml) at screening (local laboratory)
- •Current bleeding due to AHA at the time of screening
- •Plan to be adherent to emicizumab prophylaxis during the study
- •For women of childbearing potential who meet the following criteria:
- •Refrain from heterosexual intercourse or use contraceptive methods that result in a failure rate of <1% per year during the study period A woman with ≥ 12 continuous months of amenorrhea with no identified cause other than menopause and has not undergone surgical sterilization (removal of ovaries and/or uterus). use of combined oral or injected hormonal contraceptive, bilateral tubal ligation, male sterilization, hormone- releasing intrauterine devices, and copper intrauterine devices.
排除标准
- •Congenital hemophilia A
- •Treatment with aPCC within the last 24 hours before first study treatment or planned treatment with aPCC during the course of the study
- •Known positive lupus anticoagulant at the time of screening
- •Severe uncontrolled infection at the time of screening
- •Signs of active disseminated intravascular coagulation at the time of screening -
- •Emicizumab ⎯ AHA Emi Version 1.0 20
- •Current treatment for thromboembolic disease or signs of current thromboembolic disease at time of screening
- •Patients who are at high risk for TMA (e.g., have a previous medical or family history of TMA), in the investigator's judgment
- •Known severe congenital or acquired thrombophilia
- •Life expectancy <3 months at the time of screening
- •Other conditions that substantially increase risk of bleeding or thrombosis by the discretion of the investigator
- •Contraindications according to the Investigator's Brochure of emicizumab
- •Current treatment with emicizumab at time of screening
- •History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection by the discretion of the investigator
- •Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose additional risk, or would, in the opinion of the local investigator, preclude the patient's safe participation in and completion of the study
- •Addiction or other diseases that preclude the patient from appropriately assessing the nature and scope as well as possible consequences of the clinical study by the discretion of the investigator
- •Pregnant or breast-feeding women
- •Would refuse treatment with blood or blood products, if necessary.
- •Subject is in custody by order of an authority or a court of law
- •Treatment with any of the following:
- •An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 half-lives of last drug administration before Study Day 1
- •A non-hemophilia-related investigational drug within the last 30 days or 5 half-lives- before Study Day 1, whichever is longer
- •An investigational drug concurrently
- •History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
研究组 & 干预措施
Experimental-treatment
Experimental
Treatment with emicizumab
干预措施: emicizumab (Drug)
结局指标
主要结局
Primary Outcome Meassure
时间窗: 12 weeks
Number of clinically significant bleeds after 12 weeks of study drug (emicizumab)
次要结局
- Incidence and severity of adverse events(duration of entire study)
- Days of treatment with additional hemostatic agent(12 weeks)
- Remission Rate(1 to 24 weeks)
- Hospitalizations(12 weeks)
- Total dose of additional hemostatic agent(12 weeks)
研究者
Rebecca Kruse-Jarres
Professor, School of Medicine: Hematology
University of Washington
研究点 (16)
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