NCT05605678终止不适用
A Global, Non-interventional Study to Prospectively Evaluate Bleeding Episodes and Treatment Use in Patients with Hemophilia
适应症
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- ApcinteX Ltd
- 入组人数
- 108
- 试验地点
- 44
- 主要终点
- Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode
研究概览
简要总结
The primary objective of this study is to obtain prospective baseline documentation of annualized bleeding rates (ABRs) and treatment under standard-of-care (SOC) therapy among participants with hemophilia A or B. Participants in the study may be eligible to enroll in future planned interventional studies to be conducted by Sponsor.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 12 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male participants greater than or equal to (>=) 12 and less than or equal to (<=) 65 years of age
- •Who are capable of providing written informed consent (adolescent assent and parental/guardian consent when appropriate) for participation after reading the information and consent form and having the opportunity to discuss the study with the Investigator or their designee
- •With historically documented severe HemA (defined as factor VIII [FVIII] less than (<) 0.01 International Units per milliliter (IU/mL) [<1 percent {%}]), with or without inhibitors, or moderately severe to severe HemB (defined as factor IX [FIX] <=0.02 IU/mL [<=2%]) without inhibitors. Participants must be currently included in a prophylactic treatment program or if undergoing an on-demand treatment regimen must have had >=6 documented acute bleeding episodes (spontaneous or traumatic) that required coagulation factor infusion during the 6 months before enrollment OR: Historically documented HemB (defined as FIX <=0.05 IU/mL [<=5%]) with inhibitors with a historical or ongoing high titer inhibitor [>=5 Bethesda Units/mL] based on medical records or laboratory reports) and an ABR of >=6 in the 6 months before enrollment
- •Who are able to use a diary to document bleeding events and associated treatment
排除标准
- •With known thrombophilia
- •With body weight greater than (>)150 kilogram (kg) or body mass index >40
- •With known current inadequate hematologic function (eg, platelet count <100,000 per microliter [/mcL] and/or hemoglobin level <10 grams per deciliter [g/dL], <100 g/L), hepatic function (that is, total bilirubin >1.5*upper limit of normal [ULN] [excluding Gilbert syndrome], aspartate transferase and/or alanine aminotransferase levels >3*ULN; clinical signs or known laboratory or radiographic evidence consistent with cirrhosis of the liver) or renal function (that is, serum creatinine >2*ULN; based on medical records or available laboratory reports)
- •With previous deep vein thrombosis and pulmonary embolism, myocardial infarction, or stroke
- •With history of intolerance to subcutaneous injections
- •With known current uncontrolled hypertension (systolic blood pressure >160 millimeter of mercury (mm Hg); diastolic blood pressure >100 mm Hg; based on medical records)
- •With active cancer or requires therapy for cancer, except for basal cell carcinoma
- •With concurrent participation in an interventional clinical trial
- •With current or planned use of emicizumab
- •With prior, ongoing, or planned treatment with gene therapy for hemophilia
- •With history of or other evidence of recent alcohol or drug abuse as determined by the Investigator or their designee (in the 12 months before enrollment)
- •With known Human Immunodeficiency Virus (HIV) infection with CD4 count (or T-cell count) of <200 cells/mcL within 24 weeks before enrollment
- •With current or planned treatment with anticoagulant or antiplatelet drugs
- •With any other significant conditions or comorbidities that, in the opinion of the Investigator or their designee, would make the participant unsuitable for enrollment
结局指标
主要结局
Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode
时间窗: Minimum 12 weeks
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds
时间窗: Minimum 12 weeks
ABR for treated bleeds and all bleeds at the participant and bleed level (excluding bleeding episodes during surgery and/or procedures).
次要结局
未报告次要终点
研究者
研究点 (44)
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