CTRI/2023/01/049204尚未招募不适用
A Global, Non-interventional Study to Prospectively Evaluate Bleeding Episodes and Treatment Use in Patients With Hemophilia
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- ApcinteX Ltd
- 入组人数
- 200
- 试验地点
- 3
- 主要终点
- Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds
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
入排标准
- 年龄范围
- 12.00 Year(s) 至 65.00 Year(s)（—）
- 性别
- Male
入选标准
- •Male patients ≥12 and ≤65 years of age
- •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
- •Historically documented severe HemA (defined as FVIII <0.01 IU/mL [<1%]), with or without inhibitors, or moderately severe to severe HemB (defined as FIX ≤0.02 IU/mL [≤2%]) without inhibitors.
- •Patients 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
- •Able to use a diary to document bleeding events and treatment.
排除标准
- •Known thrombophilia
- •Body weight >150 kg or body mass index >40
- •Known current inadequate hematologic function (eg, platelet count <100,000/uL and/or hemoglobin level <10 g/dL, <100 g/L), hepatic function (ie, total bilirubin >1.5 x upper limit of normal [ULN] [excluding Gilbert syndrome], aspartate transferase and/or alanine aminotransferase levels >3 x ULN; clinical signs or known laboratory or radiographic evidence consistent with cirrhosis of the liver) or renal function (ie, serum creatinine >2 x ULN; based on medical records or available laboratory reports)
- •Previous deep vein thrombosis and pulmonary embolism, myocardial infarction, or stroke
- •History of intolerance to subcutaneous injections
- •Known current uncontrolled hypertension (systolic blood pressure >160 mm Hg; diastolic blood pressure >100 mm Hg; based on medical records)
- •Active cancer or requires therapy for cancer, except for basal cell carcinoma
- •Concurrent participation in an interventional clinical trial
- •Current or planned use of emicizumab
- •Prior, ongoing, or planned treatment with gene therapy for hemophilia
- •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)
- •Known HIV infection with CD4 count (or T-cell count) of <200 cells/μL within 24 weeks before enrollment
- •Current or planned treatment with anticoagulant or antiplatelet drugs
- •Any other significant conditions or comorbidities that, in the opinion of the Investigator.
结局指标
主要结局
Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds
时间窗: Minimum 12 weeks
次要结局
- Average Dose (IU/kg) of Associated Treatment Received for Bleeding Episodes per Hemophilia Product per Time Period and per Bleeding Episode(Minimum 12 weeks)
研究者
研究点 (3)
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