Goal Attainment and Physical Activity in People With Hemophilia A

Studying Preventive Treatment in Patients with Hemophilia A

Recruiting
12 years - 50 years
All
Phase N/A
35 participants needed
1 Location

Study Overview

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

You may be eligible for this study if you meet the following criteria:

  • Conditions: Hemophilia A
  • Age: 12 years - 50 years
  • Gender: All

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.

This study investigates the impact of starting prophylaxis on physical activity and goal attainment in individuals with hemophilia A. This observational study will collect data from participants in the US and Japan who are newly starting prophylaxis treatment.

Participants will be required to use a physical activity tracker compatible with their smartphone to monitor their activity levels. They will also complete electronic patient-reported outcomes and diary assessments to document their experiences and progress.

  • Who can participate: Individuals aged 12 to 50 years with moderate or severe hemophilia A who are beginning preventive treatment with an investigational medication are eligible. Participants must have access to a compatible smartphone and internet for activity tracking and reporting.
  • Study details: Participants will use a smartphone-compatible activity tracker and complete electronic assessments to report their physical activity and goal progress. An observational study means no changes will be made to their usual care.
Updated on 25 Sep 2025. Study ID: NCT06530030

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