Clinical Trial

Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis

Study acronym: STEP
Recruiting
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Summary
Hemophilia A (HA) is a genetic bleeding disorder resulting from a deficiency or absence of factor VIII (FVIII), which is necessary in the clotting process. This disorder occurs mostly in males and in severe cases causes frequent bleeding episodes in joints and muscles which can lead to progressive damage that affects mobility and quality of life. Prophylactic FVIII administered intravenously every other day has been the standard of care treatment for HA for the past few decades. Sports and physical activity are generally encouraged in patients with hemophilia on appropriate prophylactic treatment to increase strength, prevent or decrease obesity, accrue and maintain bone density and encourage normal socialization. To ensure safety with participation in sports in persons with hemophilia A (PWHA), timing of FVIII administration is often adjusted to maximize FVIII at the time of sports. The exact factor level that is needed to safely participate in sports and minimize bleeding risk is not yet known. Based on clinical practice, infusion of FVIII to near the lower limit of normal right before participation in sports generally works to prevent bleeding. The study is looking at how well the newly approved medication Emicizumab works compared to Factor VIII to prevent bleeding in patients with Hemophilia A who play sports. The study will enroll children and adolescents who are already on Emicizumab or Factor VIII who are currently playing sports.
Protocol Amendment History 8 amendments
This ClinicalTrials.gov record has been amended 8 times since 2021-08-23; most recent amendment 2026-06-30.
Status change: Not Yet Recruiting → Recruiting 2023-09-20
Trial Details
NCT Number NCT05022459
Lead Sponsor Wayne State University
Collaborators: Genentech, Inc.
Conditions Hemophilia A
Enrollment 72 participants
Start Date 2023-08-16
Primary Completion 2029-01 (estimated)
Study Completion 2029-03 (estimated)
Updated on ClinicalTrials.gov 2026-07-02