Hemophilia A
Conditions
Brief 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.
Interventions
All treatment decisions must be made independent of study participation and study-related visits/assessments are not intended to inform treatment decisions. No study medication is provided as part of study participation.
Sponsors
Sanofi
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to 50 Years
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of total goals achieved | Month 3 | Reported as number of individual goals with a raw GAS score of 0, +1, or +2 divided by the total number of goals set |
| Proportion of patients who reached a +2 or +1 on at least one goal | Month 3 | Each goal will be ranked in a 5-point Likert scale ranging from +2 to -2 with +2 indicating a better goal achievement. |
| Overall GAS score | Month 3 | 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 | Baseline | Categories of the goals selected from the goal inventory |
| Monthly average of daily step count | 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 activity count | 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 Metabolic equivalent of tasks (MET) | From Baseline to Month 12 | Estimated by the physical activity tracker |
| Monthly average of daily Moderate and Vigorous Physical Activity (MVPA) | 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 | 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 | From Baseline to Month 12 | — |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in PROMIS Pain intensity score | From Baseline to Month 12 | 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. |
| Change in HAL Score | From Baseline to Month 12 | 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) |
| Change in PROMIS pediatric Pain intensity score | From Baseline to Month 12 | 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. |
| Change in PedHAL Score | From Baseline to Month 12 | 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). |
| Change in school/work hours missed as assessed by WPAI+CIQ:HS | From Baseline to Month 12 | 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. |
| Change in productivity as assessed by WPAI+CIQ:HS | From Baseline to Month 12 | 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. |
| Change in impact on other activities as assessed by WPAI+CIQ:HS | From Baseline to 12 months | 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. |
| Mean change in total and joint domain scores of HJHS | From Baseline to 12 months | 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). |
| Monthly total and average of daily time spent in different intensity levels | From Baseline to Month 12 | Physical activity intensity defined as sedentary behavior, light intensity, moderate intensity, and vigorous intensity, |
| Monthly average of daily median activity count overall among moderate activities and among vigorous activities | From Baseline to Month 12 | Daily median time spent in moderate and vigorous physical activity will be averaged across the month |
| 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 | 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 totaled across the month. |
| Monthly total MET | From Baseline to Month 12 | — |
| Monthly total moderate or vigorous physical activity (MVPA) | From Baseline to Month 12 | Time spent in MVPA will be totaled across the month. |
| Weekly average of daily step count | From Baseline to Month 12 | — |
| Weekly average of daily activity count | 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 week. |
| Weekly average of daily MET | From Baseline to Month 12 | — |
| Weekly average of daily MVPA | From Baseline to Month 12 | Time spent in MVPA will be averaged across the week. |
| 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) | — |
Countries
United States
Outcome results
None listed