Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies (TG-INSIGHT With Joint POCUS)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 2
- 主要终点
- Hemophilia Joint Health Score
研究概览
简要总结
This is an observational research study to find out if there is a difference in the way children with moderate or severe hemophilia A, treated on two different types of factor replacement, form a clot and also evaluate if they develop tiny bleeds within the joint and subsequently early joint changes when receiving extended half-life factor VIII.
详细描述
This research study aims to compare the differences in the way children with moderate or severe hemophilia A respond when being treated with prophylactic extended half-life factor VIII based replacement therapy (such as Altuviiio) vs non-FVIII based replacement therapy (such as Hemlibra) as part of their hemophilia treatment to prevent spontaneous joint bleeds. This study will collect information about the patient's joint health, patient outcomes (using questionnaires), and laboratory testing at specific time points over 3 years.
The study will use an overall approach of comparative effectiveness research (CER) methodology. The investigators plan to recruit participants with severe (FVIII <1%) and moderate (FVIII 1-4%) HA who take extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy as their clinically prescribed treatment. Both are standard of care treatment for persons with Hemophilia A (PwHA). Using CER principals, this study will analyze differences in 1) hemostatic potential and annualized bleeding rates (Aim 1-3) and 4) joint health (structural and functional) using the Hemophilia Joint Health Score (HJHS) and imaging markers to identify subclinical bleeding and early joint tissue changes in PwHA on novel FVIII and non-FVIII therapies already in use in practice (Aim 4). The proposed study will be the first systematic observational comparative effectiveness study in PwHA on two conventional, standard-of-care novel therapies with differing mechanisms of action to evaluate the overall effectiveness of treatment.
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
- 6 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants with moderate or severe HA who are on prophylaxis with either weekly, biweekly, or every 4-weeks emicizumab or weekly efanesoctocog alfa for at least 2 months.
- •>6 months of age
排除标准
- •Participants with active FVIII inhibitor (>0.5 BU/mL)
- •Presence of an additional bleeding disorder other than hemophilia A
研究组 & 干预措施
Participants with hemophilia A
The study plans to recruit participants with severe (FVIII <1%) and moderate (FVIII 1-4%) HA.
干预措施: Half-life factor VIII based replacement therapy (Drug)
Participants with hemophilia A
The study plans to recruit participants with severe (FVIII <1%) and moderate (FVIII 1-4%) HA.
干预措施: Non-FVIII based replacement therapy (Drug)
结局指标
主要结局
Hemophilia Joint Health Score
时间窗: Obtained at baseline and every 6 months, until 3 years
To obtain and compare HJHS in patients on efanesoctocog alfa or emicizumab
Thrombin Generation
时间窗: Thrombin generation assay will be measured at 5 time points within a week of receiving dose of medication
To determine and compare in vivo thrombin generation parameters; endogenous thrombin potential (ETP) and peak thrombin (PT) for patients on efanesoctocog alfa or emicizumab
Hemophilia Joint Health Score
时间窗: Obtained at baseline and every 6 months
To obtain and compare HJHS in patients on efanesoctocog alfa or emicizumab
次要结局
- Annualized bleeding rate(From enrollment, every 6 months, until 3 years)
- Health related quality of life - CHO-KLAT(From enrollment, every 6 months, until 3 years)
- Health related quality of life - PedHAL(From enrollment, every 6 months, until 3 years)
- Health related quality of life - CATCH(From enrollment, every 6 months, until 3 years)
- Joint Tissue Changes(From enrollment, every 6 months, until 3 years)
研究者
Jessica Garcia
Assistant Professor of Pediatrics
University of Texas Southwestern Medical Center