Clinical trial NCT06530030: Goal Attainment and Physical Activity in People With Hemophilia A
Official title: Prospective, Observational Study of the Impact of Efanesoctocog Alfa (ALTUVIIIO®) on Goal Attainment and Physical Activity in People With Moderate or Severe Hemophilia A
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hemophilia A
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2024-07-31
Enrollment: 35
Sex: ALL
Minimum age (years): 12
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with moderate or severe Hemophilia A

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Proportion of total goals achieved
timeFrame: Month 3
measure: Proportion of total goals achieved
timeFrame: Month 6
measure: Proportion of total goals achieved
timeFrame: Month 9
measure: Proportion of total goals achieved
timeFrame: Month 12
measure: Proportion of patients who reached a +2 or +1 on at least one goal
timeFrame: Month 3
measure: Proportion of patients who reached a +2 or +1 on at least one goal
timeFrame: Month 6
measure: Proportion of patients who reached a +2 or +1 on at least one goal
timeFrame: Month 9
measure: Proportion of patients who reached a +2 or +1 on at least one goal
timeFrame: Month 12
measure: Overall GAS score
timeFrame: Month 3
measure: Overall GAS score
timeFrame: Month 6
measure: Overall GAS score
timeFrame: Month 9
measure: Overall GAS score
timeFrame: Month 12
measure: Frequency of selection of category of physical activity
timeFrame: Baseline
measure: Monthly average of daily step count
timeFrame: From Baseline to Month 12
measure: Monthly average of daily activity count
timeFrame: From Baseline to Month 12
measure: Monthly average of daily Metabolic equivalent of tasks (MET)
timeFrame: From Baseline to Month 12
measure: Monthly average of daily Moderate and Vigorous Physical Activity (MVPA)
timeFrame: From Baseline to Month 12
measure: Monthly average of daily intensity gradient
timeFrame: From Baseline to Month 12
measure: Monthly average of weekly intensity gradient
timeFrame: From Baseline to Month 12
Secondary outcomes:
measure: Change in PROMIS Pain intensity score
timeFrame: From Baseline to Month 12
measure: Change in HAL Score
timeFrame: From Baseline to Month 12
measure: Change in PROMIS pediatric Pain intensity score
timeFrame: From Baseline to Month 12
measure: Change in PedHAL Score
timeFrame: From Baseline to Month 12
measure: Change in school/work hours missed as assessed by WPAI+CIQ:HS
timeFrame: From Baseline to Month 12
measure: Change in productivity as assessed by WPAI+CIQ:HS
timeFrame: From Baseline to Month 12
measure: Change in impact on other activities as assessed by WPAI+CIQ:HS
timeFrame: From Baseline to 12 months
measure: Mean change in total and joint domain scores of HJHS
timeFrame: From Baseline to 12 months
measure: Monthly total and average of daily time spent in different intensity levels
timeFrame: From Baseline to Month 12
measure: Monthly average of daily median activity count overall among moderate activities and among vigorous activities
timeFrame: From Baseline to Month 12
measure: Monthly average of daily median MET overall among moderate activities and among vigorous activities
timeFrame: From Baseline to Month 12
measure: Monthly average of weekly power law exponent alpha
timeFrame: From Baseline to Month 12
measure: Monthly average of median bout length and percent of time of bout length > median bout length for each intensity level
timeFrame: From Baseline to Month 12
measure: Monthly average of weekly 80th, 90th, 95th, and 100th percentiles of activity count
timeFrame: From Baseline to Month 12
measure: Monthly average of weekly 80th, 90th, 95th, and 100th percentiles of MET
timeFrame: From Baseline to Month 12
measure: Monthly total step count
timeFrame: From Baseline to Month 12
measure: Monthly total activity count
timeFrame: From Baseline to Month 12
measure: Monthly total MET
timeFrame: From Baseline to Month 12
measure: Monthly total moderate or vigorous physical activity (MVPA)
timeFrame: From Baseline to Month 12
measure: Weekly average of daily step count
timeFrame: From Baseline to Month 12
measure: Weekly average of daily activity count
timeFrame: From Baseline to Month 12
measure: Weekly average of daily MET
timeFrame: From Baseline to Month 12
measure: Weekly average of daily MVPA
timeFrame: From Baseline to Month 12
measure: Weekly average of daily intensity gradient
timeFrame: From Baseline to Month 12
measure: Weekly intensity gradient
timeFrame: From Baseline to Month 12
measure: Number of participants with AE, SAEs, AESIs
timeFrame: Baseline to end of study (12 months)
measure: Number of participants with AEs, SAEs, AESIs related to efanesoctocog alfa
timeFrame: Baseline to end of study (12 months)
measure: Proportion of total planned doses that were missed
timeFrame: From baseline to end of study (12 months)
measure: Average time between treatment dose and physical activity
timeFrame: From baseline to end of study (12 months)
measure: Number of patients with no reported bleeds requiring treatment
timeFrame: From baseline to end of study (12 months)
measure: Average number of per patient treatment doses administered for treatment of bleeds
timeFrame: From baseline to end of study (12 months)
Other outcomes:
Locations:
city: Peoria
country: United States
facility: Bleeding and Clotting Disorders Institute
state: Illinois
Study officials:
References: