Goal Attainment and Physical Activity in People With 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

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.

Study Overview

• Status: Terminated
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: efanesoctocog alfa

• Study Type: Observational
• Enrollment (Actual): 3

Contacts and Locations

Study Locations

• United States
  • Illinois
    • Peoria, Illinois, United States, 61614
      • Bleeding and Clotting Disorders Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with moderate or severe Hemophilia A

Description

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/Exclusion criteria may apply. The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort; patients aged 12 to 50 years with moderate or severe hemophilia A who are newly starting prophylaxis with efa	Drug: efanesoctocog alfa; 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.; Other Names: Altuviiio

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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

Outcome Measure	Measure Description	Time Frame
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)

Collaborators and Investigators

• Sponsor: Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): November 26, 2024
• Primary Completion (Actual): September 18, 2025
• Study Completion (Actual): September 18, 2025

Study Registration Dates

• First Submitted: July 17, 2024
• First Submitted That Met QC Criteria: July 26, 2024
• First Posted (Actual): July 31, 2024

Study Record Updates

• Last Update Posted (Actual): January 20, 2026
• Last Update Submitted That Met QC Criteria: January 15, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia A
• Other Study ID Numbers: OBS18140; U1111-1306-7241 (Other Identifier: WHO ICTRP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• product manufactured in and exported from the U.S.: No