NCT07789587尚未招募不适用
Efficacy and Safety of Bometase Alfa for the Treatment of Bleeding in Acquired Hemophilia A: A Prospective, Single-Arm, Exploratory Study
Institute of Hematology & Blood Diseases Hospital, China1 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2026年8月1日最近更新:
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Incidence of effective hemostasis rate at 8 hours after the first administration
研究概览
简要总结
This is a single-center, prospective, single-arm, exploratory study designed to evaluate the efficacy and safety of Bometase Alfa for the on-demand treatment of bleeding episodes in patients with acquired hemophilia A. A total of 20 patients with acquired hemophilia A experiencing bleeding events will be enrolled. Bometase Alfa will be administered at 0.1 U/kg for non-severe bleeding and 0.16 U/kg for severe bleeding, with consecutive doses given at 4-hour intervals until hemostasis is achieved. Treatment will be discontinued once hemostasis is achieved or if symptoms suggestive of arterial thrombosis occur, followed by a safety assessment. Rescue therapy will be initiated if bleeding remains uncontrolled after three consecutive administrations for a single bleeding episode, or if bleeding continues to worsen during treatment and the investigator determines that further treatment with Bometase Alfa is unlikely to provide clinical benefit and may pose a medical risk.
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
- •Age ≥18 years;
- •Confirmed diagnosis of acquired hemophilia A, meeting the following criteria:
- •Isolated prolongation of activated partial thromboplastin time (APTT) with a normal prothrombin time (PT);
- •Reduced factor VIII coagulant activity (FVIII:C <50%);
- •Positive FVIII inhibitor, defined as ≥0.6 BU/mL as measured by the Bethesda assay or Nijmegen-modified Bethesda assay, or failure of a 1:1 mixing study with normal plasma to achieve complete correction;
- •No evidence of congenital hemophilia, von Willebrand disease, or lupus anticoagulant;
- •Presence of clinically significant active bleeding, including, but not limited to, muscle or subcutaneous hematoma, gastrointestinal or genitourinary bleeding, postpartum or postoperative bleeding, or deep-seated or life-threatening organ bleeding;
- •Provision of written informed consent by the patient and/or a legally authorized representative;
- •Ability to comply with the study follow-up schedule for at least 30 da
排除标准
- •Congenital hemophilia A or B, or any other confirmed congenital coagulation factor deficiency;
- •Isolated prolonged activated partial thromboplastin time (APTT) due to lupus anticoagulant or antiphospholipid syndrome, with a negative FVIII inhibitor and normal FVIII activity; or coagulation abnormalities caused by disseminated intravascular coagulation (DIC) or severe liver disease that do not fulfill the diagnostic criteria for acquired hemophilia A;
- •A history or symptoms of any arterial or venous thromboembolic event within 3 months before enrollment, including atherosclerosis, myocardial infarction, ischemic stroke, transient ischemic attack, deep vein thrombosis, or pulmonary embolism, or the presence of DIC;
- •Use of factor VII (FVII), activated factor VII (FVIIa), tranexamic acid, or aminocaproic acid within 1 day before the planned administration of the study drug; or use of prothrombin complex concentrate (PCC) or factor VIII (FVIII) within 3 days before the first administration;
- •Female participants who are pregnant or breastfeeding, or have a positive pregnancy test;
- •Known hypersensitivity to the investigational product or any of its excipients;
- •Inability to obtain informed consent, including patients unable to express their wishes and without a legally authorized representative;
- •Inability to comply with the study follow-up requirements or investigator-determined poor compliance;
- •Any other condition for which the investigator considers the participant unsuitable for study participation.
结局指标
主要结局
Incidence of effective hemostasis rate at 8 hours after the first administration
时间窗: 8 hours
次要结局
- Rate of rescue therapy(30 days)
- Incidence of Treatment-Emergent Adverse Events (AES)(AES was assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), Version 5.0.)
- Incidence of effective hemostasis rate at 12 hours after the first administration(12 hours)
- Time to achieve clinical hemostasis(30 days)
- Amount of blood product use(30 days)
- Dose of Bometase Alfa administered(30 days)
研究者
研究点 (1)
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