Goal Attainment and Physical Activity in People With Hemophilia A
NCT Number: NCT06530030
Acronym: ACTIVIIITY
Status: NOT_YET_RECRUITING
Study Type: OBSERVATIONAL
Conditions: Hemophilia A
Interventions: DRUG: efanesoctocog alfa
Sponsor: Sanofi
Sex: ALL
Age: CHILD, ADULT
Enrollment: 35
Study Design: Observational Model: |Time Perspective: p
Start Date: 2024-08-05
Completion Date: 2026-05-08

Summary:
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.

Primary Outcome Measures: 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
Secondary Outcome Measures: 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)