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.
Participant (if 18 years of age or older) or parent/LAR is willing and able to provide written informed consent; minor participant is willing and able to provide assent, if applicable based on site and local regulations
Males and females between 6 to ≤ 19 years of age at time of enrollment with moderate to severe Hemophilia A (FVIII activity ≤ 5%) without inhibitors are eligible for participation in this study
Participants must be on Emicizumab or standard FVIII prophylaxis per institutional/primary hematologist recommendations
Participants must be engaging in or registered to start participating in one or more sports activities with moderate to high risk of bleeding as defined by the NHF- Playing it Safe guidelines (numerical rating >/= 2).
Participant/parent/LAR unwilling to provide informed consent/assent
Unwilling to log or document bleeds and treatment information as per study guidelines
Participants with any other bleeding disorders will be excluded
Patients who are pregnant, planning to become pregnant, or breastfeeding should not be enrolled in the study
This study will enroll patients who are already on Emicizumab or FVIII prophylaxis as per standard of care and who are already participating in sports activities with moderate to high risk of bleeding.
Cohort
Prospective
Emicizumab
DrugPatients in this group will be on standard of care Emicizumab prophylaxis for Hemophilia A
FVIII
DrugPatients in this group will be on standard of care FVIII prophylaxis for Hemophilia A
Number of bleeds
Number of bleeds without trauma, during participation in sports activities
Change in HJHS scores
Change in Hemophilia Joint Health Scores (HJHS). The HJHS provides a total score (higher score is worse; max=124), joint specific scores, and a global gait score.
Change in bone mineral density
Change in bone mineral density as measured by DEXA Scan
Change in biomarkers of bone and joint health
Change in biomarkers of bone and joint health as measured by (sRANKL (osteoclastogenesis), CTX-1 (Bone resorption), CS846 (cartilage repair) and IL-1 beta, IL6 and TNF-alpha (markers of inflammation).
Number of FVIII doses for breakthrough bleeding episodes
Number of doses of factor needed to treat breakthrough bleeding episodes
Click on the lead sponsor to view all of their trials.
Wayne State University
Lead sponsor
Genentech, Inc.
Collaborator
