Prospective, Observational Study of the Impact of Efanesoctocog Alfa (ALTUVIIIO®) on Goal Attainment and Physical Activity in People With Moderate or Severe Hemophilia A
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- Sanofi
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Proportion of total goals achieved
研究概览
简要总结
This is a Phase 4, multi-center, observational study conducted in patients aged 12 to 50 years with moderate or severe hemophilia A who are newly starting prophylaxis with efa in the US and Japan.
This study aims to enroll 35 patients.
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
- 12 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •At enrollment, newly starting prophylaxis therapy with efanesoctocog alfa according to usual clinical practice, with efanesoctocog alfa 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 activity tracking device
排除标准
- •Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥ 0.60 BU/mL
- •Use of efanesoctocog alfa for prophylaxis in the 6 months prior to enrollment
- •NOTE: Other Inclusion/Exclusion criteria may apply. The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
研究组 & 干预措施
Cohort
patients aged 12 to 50 years with moderate or severe hemophilia A who are newly starting prophylaxis with efa
干预措施: efanesoctocog alfa (Drug)
结局指标
主要结局
Proportion of total goals achieved
时间窗: Month 12
Proportion of patients who reached a +2 or +1 on at least one goal
时间窗: Month 12
Overall GAS score
时间窗: Month 12
Goal Attainment Scaling (GAS) is a patient-centric clinician-reported outcome measure utilized to select and scale goals, with higher scores indicating greater goal attainment. An overall GAS score utilizing all goals set by a patient at each time point of interest will be calculated.
Frequency of selection of category of physical activity
时间窗: Baseline
Categories of the goals selected from the goal inventory
Monthly average of daily step count
时间窗: From Baseline to Month 12
The monthly average of daily step count (recorded by physical tracker) will be calculated using the total steps recorded in a 30-day period, accounting for the number of compliant days in that period.
Monthly average of daily Metabolic equivalent of tasks (MET)
时间窗: From Baseline to Month 12
Estimated by the physical activity tracker
Monthly average of daily activity count
时间窗: From Baseline to Month 12
Activity count includes: time spent in x, y, and z axes, vector magnitude counts, estimated calories, wear time, time spent in locomotion and non-locomotion. Counts will be averaged across the month.
Monthly average of daily Moderate and Vigorous Physical Activity (MVPA)
时间窗: From Baseline to Month 12
Summarized from MET data with time spent in moderate intensity, and vigorous intensity physical activity
Monthly average of daily intensity gradient
时间窗: From Baseline to Month 12
The intensity gradient is a measure of activity intensity distribution that reflects the distribution of intensity of physical activity by modeling the relationship between intensity and time.
Monthly average of weekly intensity gradient
时间窗: From Baseline to Month 12
次要结局
- Change in HAL Score(From Baseline to Month 12)
- Change in school/work hours missed as assessed by WPAI+CIQ:HS(From Baseline to Month 12)
- Change in PROMIS Pain intensity score(From Baseline to Month 12)
- Change in PROMIS pediatric Pain intensity score(From Baseline to Month 12)
- Change in PedHAL Score(From Baseline to Month 12)
- Monthly total and average of daily time spent in different intensity levels(From Baseline to Month 12)
- Monthly average of daily median activity count overall among moderate activities and among vigorous activities(From Baseline to Month 12)
- Monthly average of weekly power law exponent alpha(From Baseline to Month 12)
- Mean change in total and joint domain scores of HJHS(From Baseline to 12 months)
- Change in productivity as assessed by WPAI+CIQ:HS(From Baseline to Month 12)
- Change in impact on other activities as assessed by WPAI+CIQ:HS(From Baseline to 12 months)
- Monthly average of weekly 80th, 90th, 95th, and 100th percentiles of activity count(From Baseline to Month 12)
- Monthly average of daily median MET overall among moderate activities and among vigorous activities(From Baseline to Month 12)
- Number of patients with no reported bleeds requiring treatment(From baseline to end of study (12 months))
- Monthly average of median bout length and percent of time of bout length > median bout length for each intensity level(From Baseline to Month 12)
- Monthly average of weekly 80th, 90th, 95th, and 100th percentiles of MET(From Baseline to Month 12)
- Monthly total step count(From Baseline to Month 12)
- Monthly total activity count(From Baseline to Month 12)
- Monthly total MET(From Baseline to Month 12)
- Weekly average of daily intensity gradient(From Baseline to Month 12)
- Number of participants with AE, SAEs, AESIs(Baseline to end of study (12 months))
- Number of participants with AEs, SAEs, AESIs related to efanesoctocog alfa(Baseline to end of study (12 months))
- Proportion of total planned doses that were missed(From baseline to end of study (12 months))
- Average time between treatment dose and physical activity(From baseline to end of study (12 months))
- Monthly total moderate or vigorous physical activity (MVPA)(From Baseline to Month 12)
- Weekly average of daily step count(From Baseline to Month 12)
- Weekly average of daily activity count(From Baseline to Month 12)
- Weekly average of daily MET(From Baseline to Month 12)
- Weekly average of daily MVPA(From Baseline to Month 12)
- Weekly intensity gradient(From Baseline to Month 12)
- Average number of per patient treatment doses administered for treatment of bleeds(From baseline to end of study (12 months))