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

Conditions

• Hemophilia A

Eligibility Criteria

Sex

ALL

Age

6 Years - 19 Years

Healthy Volunteers

Not accepted

Interventions

• Emicizumab — DRUG
  Patients in this group will be on standard of care Emicizumab prophylaxis for Hemophilia A
• FVIII — DRUG
  Patients in this group will be on standard of care FVIII prophylaxis for Hemophilia A

Study Details

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.

Key Dates

Start date

Aug 16, 2023

Status verified

Jan 2026

Primary completion

Jan 31, 2029

Completion

Mar 31, 2029

Study Design

Enrollment

72 participants (estimated)

Arms

• Arm: Emicizumab Prophylaxis
  This group will include patients on standard of care Emicizumab prophylaxis for Hemophilia A
• Arm: FVIII Prophylaxis
  This group will include patients on standard of care FVIII prophylaxis for Hemophilia A

Primary Outcome Measure

Number of bleeds [ Time Frame: 2 years ]

Central Contacts

• Amy Stolinski, MPH
  313-577-7500
  [email protected]

Locations (9)

Find similar trials in Little Rock, AR

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