Hemophilia A efanesoctocog alfa : recombinant Factor VIII
Conditions
Interventions
None listed
Sponsors
Kidani Yoko
Eligibility
Inclusion criteria
Inclusion criteria: 1. 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 2. At enrollment, newly starting prophylaxis therapy with efa according to usual clinical practice, with efa initiated within 6 months after the enrollment visit. 3. Diagnosis of moderate (endogenous FVIII activity between 1% to 5% of normal) or severe (endogenous FVIII activity <1% of normal) hemophilia A 4. Aged 12 to 50 years at time of enrollment, inclusive. 5. 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. 6. Availability of home-based access to internet for electronic patient-reported outcome (ePRO)/diary assessments. 7. Willingness to utilize the ActiGraph LEAP provisional device.
Exclusion criteria
Exclusion criteria: 1. Enrollment in another concurrent interventional clinical study, or intake of an investigational medicinal product within 3 months prior to inclusion in this study. 2. Current diagnosis of a FVIII inhibitor, defined as inhibitor titer >=0.60 BU/mL. 3. Use of efa for prophylaxis in the 6 months prior to enrollment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Proportion of total goals achieved at 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 - 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) - Monthly average of weekly intensity gradient (intercept and slope) | — |
Secondary
| Measure | Time frame |
|---|---|
| - Among participants 18 years and older: - Change from baseline in PROMIS Pain intensity score - Change from baseline in HAL score - Among participants 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 | — |
Countries
Japan, US
Contacts
Public ContactWaka Shimomoto
IQVIA Services Japan G.K.
Outcome results
None listed