治験レーダー

Proportion of total goals achieved

Reported as number of individual goals with a raw GAS score of 0, +1, or +2 divided by the total number of goals set

Month 3

Proportion of total goals achieved

Month 6

Proportion of total goals achieved

Month 9

Proportion of total goals achieved

Month 12

Proportion of patients who reached a +2 or +1 on at least one goal

Each goal will be ranked in a 5-point Likert scale ranging from +2 to -2 with +2 indicating a better goal achievement.

Month 3

Proportion of patients who reached a +2 or +1 on at least one goal

Month 6

Proportion of patients who reached a +2 or +1 on at least one goal

Month 9

Proportion of patients who reached a +2 or +1 on at least one goal

Month 12

Overall GAS score

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.

Month 3

Overall GAS score

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.

Month 6

Overall GAS score

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.

Month 9

Overall GAS score

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.

Month 12

Frequency of selection of category of physical activity

Categories of the goals selected from the goal inventory

Baseline

Monthly average of daily step count

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.

From Baseline to Month 12

Monthly average of daily activity count

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.

From Baseline to Month 12

Monthly average of daily Metabolic equivalent of tasks (MET)

Estimated by the physical activity tracker

From Baseline to Month 12

Monthly average of daily Moderate and Vigorous Physical Activity (MVPA)

Summarized from MET data with time spent in moderate intensity, and vigorous intensity physical activity

From Baseline to Month 12

Monthly average of daily intensity gradient

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.

From Baseline to Month 12

Monthly average of weekly intensity gradient

From Baseline to Month 12

Change in PROMIS Pain intensity score

Patient-Reported Outcome Measurement Information System (PROMIS): assessment of pain over the prior 7-day period (2 of the 3 questions) and right now (1 of the 3 questions), with patients rating their pain on a scale of 0 (had no pain) to 5 (very severe). Total score ranges from 0 to 15 and a higher score indicates worst pain intensity.

From Baseline to Month 12

Change in HAL Score

Hemophilia Activities List (HAL) is a 42-item questionnaire to access patient ability across 7 domains: lying down/ sitting/ kneeling/ standing (8 items), functions of the legs (9 items), functions of the arms (4 items), use of transportation (3 items), self-care (5 items), household tasks (6 items), and leisure activities (7 items). Patients rate each question on a Likert scale ranging from 1 (impossible) to 6 (never). Several items have a "not applicable" option as well. There is also a visual analog scale allowing patients to self-report an overall score. A normalized total score (sum of all normalized domains) ranges from 0 (the worst possible functional status) to 100 (the best possible functional status)

From Baseline to Month 12

Change in PROMIS pediatric Pain intensity score

Assessment of the average pain over the prior 7-day period, with patients rating their pain on a scale of 0 (no pain) to 10 (worst pain they can think of). A higher score indicates worst pain intensity.

From Baseline to Month 12

Change in PedHAL Score

Pediatric Hemophilia Activities List (PedHAL) is a 53-item questionnaire to access patient functional ability across 7 domains: lying down/ sitting/ kneeling/ standing (10 items), functions of the legs (11 items), functions of the arms (6 items), use of transportation (3 items), self-care (9 items), household tasks (3 items), and leisure activities and sports (11 items). Patients rate each question on a Likert scale ranging from 1 (impossible) to 6 (never). Several items have a "not applicable" option as well. There is also a visual analog scale allowing patients to self-report an overall score. A normalized total score (sum of all normalized domains) ranges from 0 (the worst possible functional status) to 100 (the best possible functional status).

From Baseline to Month 12

Change in school/work hours missed as assessed by WPAI+CIQ:HS

The Work Productivity and Activity Impairment plus Classroom Impairment Questions: Hemophilia Specific (WPAI + CIQ: HS) is a 9-item questionnaire assessing the effect of hemophilia on patients' ability to work and/or attend classes.

From Baseline to Month 12

Change in productivity as assessed by WPAI+CIQ:HS

The Work Productivity and Activity Impairment plus Classroom Impairment Questions: Hemophilia Specific (WPAI + CIQ: HS) is a 9-item questionnaire assessing the effect of hemophilia on patients' ability to perform regular daily activities.

From Baseline to Month 12

Change in impact on other activities as assessed by WPAI+CIQ:HS

The Work Productivity and Activity Impairment plus Classroom Impairment Questions: Hemophilia Specific (WPAI + CIQ: HS) is a 9-item questionnaire assessing the effect of hemophilia on patients' ability to perform other activities.

From Baseline to 12 months

Mean change in total and joint domain scores of HJHS

The Hemophilia Joint Health Score (HJHS) assesses 6 index joints (left and right elbows, knees and ankles) and a global gait. For each of the 6 joints, the following items are scored: swelling (scored 0-3), duration of swelling (0-1), muscle atrophy (0-2), crepitus on motion (0-2), flexion loss (0-3), extension loss (0-3), joint pain (0-2), and strength (0-4). The HJHS provides a total score (0-124 points with higher score indicating worse joint health), joint specific scores (0-20 points), and a global gait score (0-4 points).

From Baseline to 12 months

Monthly total and average of daily time spent in different intensity levels

Physical activity intensity defined as sedentary behavior, light intensity, moderate intensity, and vigorous intensity,

From Baseline to Month 12

Monthly average of daily median activity count overall among moderate activities and among vigorous activities

Daily median time spent in moderate and vigorous physical activity will be averaged across the month

From Baseline to Month 12

Monthly average of daily median MET 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

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 activity count

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

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 totaled across the month.

From Baseline to Month 12

Monthly total MET

From Baseline to Month 12

Monthly total moderate or vigorous physical activity (MVPA)

Time spent in MVPA will be totaled across the month.

From Baseline to Month 12

Weekly average of daily step count

From Baseline to Month 12

Weekly average of daily activity count

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 week.

From Baseline to Month 12

Weekly average of daily MET

From Baseline to Month 12

Weekly average of daily MVPA

Time spent in MVPA will be averaged across the week.

From Baseline to Month 12

Weekly average of daily intensity gradient

From Baseline to Month 12

Weekly 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)

Number of patients with no reported bleeds requiring treatment

From baseline to end of study (12 months)

Average number of per patient treatment doses administered for treatment of bleeds

From baseline to end of study (12 months)