A Prospective and Retrospective Interventional Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 45
- 试验地点
- 1
- 主要终点
- Systematic joint and bone health assessments before and after enrollment
研究概览
简要总结
Study Objectives*
- Provide a systematic evaluation of the treatment outcomes in patients with hemophilia A
- Emphasize the importance and clinical benefits of rFVIII-Fc in joint and bone health.
- Compare the clinical outcomes from 1 year before and after switching to EHL.
- Exploratory: Identify biomarkers that could provide more useful and convenient evaluations of joint and bone health. (Time-saving and easy to monitor)
详细描述
In this study, we would like to focus on the evaluation of joint and bone health in persons with severe hemophilia A (PwHA) treated with rFVIIIFc prophylaxis. We also aim to identify potential biomarkers for assessing the joint and bone health of PwHA. Since the joint and bone health examinations are time-consuming, expensive, and require special equipment, the patients could only receive the examinations annually to realize the status of their joint health. The biomarkers testing will provide a more convenient and time-saving option on the joint and bone health evaluation for PwHA. Therefore, surrogate of biochemical marker(s) to represent the joint and BMD progression is an unmet need for clinical practice. Investigating different biomarkers and combining them with the joint and BMD results to provide a systematic approach for elucidating the treatment outcomes of PwHA treated with rFVIIIFc is major goal of this study.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Patients with severe hemophilia A (all ages).
- •Patients with moderate type hemophilia A with hemophilic arthropathy.
- •Currently on rFVIIIFc regular prophylaxis and have previous medical record with regular prophylaxis with standard half-life product for one year
- •Able and willing to undergo joint and bone examinations
排除标准
- •Participants of other interventional studies.
- •Patients with current inhibitors.
- •History of major neurological disease (eg. Stroke, Parkinson's disease, neuropathy, etc.)
- •History of major psychiatric disease (eg. Schizophrenia, bipolar disorder)
- •Significantly impaired vision/hearing
结局指标
主要结局
Systematic joint and bone health assessments before and after enrollment
时间窗: up to 5 years
Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) on patients' ankles, knees, and elbows. Also evaluate the Hemophilia Joint Health Score (HJHS) and Dexa scan at the hip.
次要结局
- Serum markers(up to 5 years)
研究者
Chen Yeu-Chin
Director of the Hemophilia Center
Tri-Service General Hospital