Goal Attainment and Physical Activity in People With Hemophilia A

Studying Prophylaxis in Hemophilia A Patients

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.

Updated on 04 Apr 2025. Study ID: NCT06530030

This study investigates the use of prophylaxis treatment in patients with moderate or severe hemophilia A. Hemophilia A is a genetic disorder where the blood does not clot properly due to low levels of a protein called factor VIII. This study is being conducted in patients aged 12 to 50 years in the US and Japan who are starting a specific prophylaxis treatment for the first time.

Participants will be observed as they begin their prophylaxis treatment. They must have access to a smartphone and the internet to report their outcomes electronically. They will also use an activity tracking device to monitor their physical activity levels.

  • Who can participate: Patients aged 12 to 50 years with moderate or severe hemophilia A who are starting prophylaxis treatment for the first time can participate. They need to have access to a smartphone and the internet, and be willing to use an activity tracker.
  • Study details: Participants will be required to use a smartphone and the internet for electronic reporting of their outcomes. They will also use an activity tracking device to monitor their physical activity. This study does not involve any investigational treatments, just observation of existing treatment practices.

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