Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 4
- 主要终点
- joint health comparison
研究概览
简要总结
The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current standard of care, most patients are on prophylactic FVIII replacement therapy intravenously several times weekly with a goal of keeping the trough >1% FVIII. Recent phase 3 data suggest superior bleed protection with emicizumab prophylaxis every 1-2 weeks. It is the purpose of this study to longitudinally assess joint health and bone density over 3 years and to compare the effect of routine factor VIII prophylaxis with emicizumab prophylaxis.
详细描述
Summary: This retrospective/prospective, non-randomized controlled study, whereby patients will be included in 1 of 2 groups depending on their current treatments as outlined below. Patients will be assigned to a specific group by the clinician/PI based on their current regimen with the intent to stay on this regimen for the next 3 years.
Duration of Study and Subject Participation: Study subject enrollment is anticipated to occur over about 12 months and study duration per subject will be 3 years. Thus a total study duration of 4 years is anticipated.
Intervention: By using musculoskeletal ultrasound, the investigators will measure joint health among participants and assess joint health changes over 3 years compared between group A (factor prophylaxis) vs. group B (emicizumab prophylaxis) as assessed by MSKUS at baseline and after 3 years. Other outcomes such as bone density, comparative assessment of joint and overall health status, and exploration of potential biomarkers for joint and bone health will also be assessed.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male gender
- •Severe hemophilia A (factor VIII < 1%)
- •Age ≥ 16 year
- •Either on prophylaxis with factor VIII or emicizumab with the intention to stay on the current regimen for the next 3 years
- •Willing and able to give written informed consent/assent
- •Willing to undergo MSKUS, DEXA scan +/- collection of blood sampling for repository biomarkers
- •Willing to come in for baseline and 3 yearly visits
- •Willing to answer phone survey for bleeding and safety every 3 months
排除标准
- •Current FVIII inhibitor of > 0.6 BU
- •Unable to take FVIII replacement
- •Other known bleeding disorder
- •Other rheumatologic disorder affecting joints
- •Other known neuromotor defect (making physical exam difficult)
结局指标
主要结局
joint health comparison
时间窗: 3 years
Joint health changes over 3 years compared between group A (factor prophylaxis) vs. group B (emicizumab prophylaxis) as assessed by MSKUS at baseline and after 3 years.
次要结局
- bone density comparison(3 years)
- comparative assessment of joint and overall health(3 years)
- mid-point assessment of joint health and bone density(2 years)
- biomarkers for joint and bone health(through completion of study, average 3 years)
研究者
Rebecca Kruse-Jarres, MD, MPH
Executive/Medical Director, Washington Center for Bleeding Disorders at Washington Institute for Coagulation
Washington Institute for Coagulation