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Prospective, observational study of the impact of efanesoctocog alfa (ALTUVIIIO) on goal attainment and physical activity in people with moderate or severe Hemophilia A (ACTIVIIITY)

Prospective, observational study of the impact of efanesoctocog alfa (ALTUVIIIO) on goal attainment and physical activity in people with moderate or severe Hemophilia A (ACTIVIIITY)

Kidani Yoko

sanofi K.K.

Tokyo opera city tower,3-20-2, Nishi Shinjuku, Shinjuku-ku, Tokyo

Sanofi_ACTIVIIITY_Japan@iqvia.com

Shimomoto Waka

IQVIA Services Japan G.K.

Keikyu Dai-ichi Bldg 4-10-18, Takanawa, Minato-ku, Tokyo

+81-90-8655-6223

waka.shimomoto@iqvia.com

Complete

May. 28, 2025

Observational

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.

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.

Hemophilia A

efanesoctocog alfa : recombinant Factor VIII

D006467

- 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)

- Among participants 18 years and older:
- Change from baseline in PROMIS Pain intensity score
- Change from baseline in HAL score
- Among participants <18 years of age:
- Change from baseline in PROMIS pediatric pain intensity score
- Change from baseline in PedHAL score
- Change from baseline in school/work hours missed as assessed by WPAI+CIQ:HS
- Change from baseline in productivity as assessed by WPAI+CIQ:HS
- Change from baseline in impact on other activities as assessed by WPAI+CIQ:HS
- Mean change from baseline in total and joint domain scores of HJHS
- Monthly total and average of daily time spent in different intensity levels (sedentary behavior, light intensity, moderate intensity, and vigorous intensity)
- Monthly average of daily median activity count overall, among moderate activities, and among vigorous activities
- Monthly average of daily median MET overall, among moderate activities, and among vigorous activities
- Monthly average of weekly power law exponent alpha
- Monthly average of median bout length and percent of time of bout length > 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

+81-3-6416-1868

Dec. 18, 2024

Yes

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

US