jRCT1031250136招募中不适用
Prospective, observational study of the impact of efanesoctocog alfa (ALTUVIIIO) on goal attainment and physical activity in people with moderate or severe Hemophilia A (ACTIVIIITY)
Sanofi Aventis Recherche & Developpement0 个研究点目标入组 35 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 35
- 主要终点
- Proportion of total goals achieved at quarterly timepoints and EoS
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Observational
入排标准
- 年龄范围
- 12age old over 至 50age old under（—）
入选标准
- •Signed and dated informed consent form (ICF) by the patient, or the patients legally authorized representative(s) for patients under the legal age, before any study-related activities are undertaken. Assent should be obtained from pediatric patients according to local institutional/ institutional review board (IRB) regulations
- •At enrollment, newly starting prophylaxis therapy with efa according to usual clinical practice, with efa initiated within 6 months after the enrollment visit.
- •Diagnosis of moderate (endogenous FVIII activity between 1% to 5% of normal) or severe (endogenous FVIII activity <1% of normal) hemophilia A
- •Aged 12 to 50 years at time of enrollment, inclusive.
- •Access to a smartphone device (Android version 12.0 or higher; or iOS 13 or higher) with Bluetooth 4.0 (minimum) or 5.0 (recommended) capabilities for compatibility with physical activity tracker.
- •Availability of home-based access to internet for electronic patient-reported outcome (ePRO)/diary assessments.
- •Willingness to utilize the ActiGraph LEAP provisional device.
排除标准
- •Enrollment in another concurrent interventional clinical study, or intake of an investigational medicinal product within 3 months prior to inclusion in this study.
- •Current diagnosis of a FVIII inhibitor, defined as inhibitor titer >=0.60 BU/mL.
- •Use of efa for prophylaxis in the 6 months prior to enrollment.
结局指标
主要结局
Proportion of total goals achieved at quarterly timepoints and EoS
时间窗: quarterly timepoints and EoS
Proportion of total goals achieved at quarterly timepoints and EoS
Overall physical activity goals (categories/type) ranked by frequency of selection
Overall physical activity goals (categories/type) ranked by frequency of selection
Monthly average of daily step count, activity count, MET, MVPA, intensity gradient (intercept and slope)
时间窗: monthly
Monthly average of daily step count, activity count, MET, MVPA, intensity gradient (intercept and slope)
Monthly average of weekly intensity gradient (intercept and slope)
时间窗: monthly
Monthly average of weekly intensity gradient (intercept and slope)
Proportion of patients who reached a +2 or +1 on at least one goal at quarterly timepoints and EoS
时间窗: quarterly timepoints and EoS
Proportion of patients who reached a +2 or +1 on at least one goal at quarterly timepoints and EoS
Overall GAS score quarterly and at EoS
时间窗: quarterly and at EoS
Overall GAS score quarterly and at EoS
次要结局
- Change from baseline in PROMIS Pain intensity score
- Change from baseline in HAL score
- Change from baseline in PROMIS pediatric pain intensity score
- Change from baseline in PedHAL score
- Change from baseline in school/work hours missed as assessed by WPAI+CIQ:HS
- Change from baseline in productivity as assessed by WPAI+CIQ:HS
- Change from baseline in impact on other activities as assessed by WPAI+CIQ:HS
- Mean change from baseline in total and joint domain scores of HJHS
- Monthly total and average of daily time spent in different intensity levels (sedentary behavior, light intensity, moderate intensity, and vigorous intensity)
- Monthly average of weekly power law exponent alpha
- Monthly average of daily median activity count overall, among moderate activities, and among vigorous activities
- Monthly average of daily median MET overall, among moderate activities, and among vigorous activities
- Monthly average of median bout length and percent of time of bout length > median bout length for each intensity level (sedentary behavior, light intensity, moderate intensity, and vigorous intensity)
- Monthly average of weekly 80th, 90th, 95th, and 100th percentiles of activity count and MET
- Monthly total step count, activity count, MET, MVPA
- Weekly average of daily step count, activity count, MET, MVPA, intensity gradient (intercept and slope)
- Weekly intensity gradient (intercept and slope)
- Total AEs, proportion of patients with at least one AE, and average number of AEs per patient
- Total SAEs, proportion of patients with at least one SAE, and average number of SAEs per patient
- Total AESIs, proportion of patients with at least one AESI, and average number of AESIs per patient
- Number of AEs, SAEs, and AESIs related to efa
- Proportion of total planned doses that were missed
- Average time between treatment dose and physical activity
- Number of patients with no reported bleeds requiring treatment
- Average number of per patient treatment doses administered for treatment of bleeds
研究者
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