Hemophilia A Clinical Trial
— STEPOfficial title:
Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis -STEP: SporTs Emicizumab Prophylaxis
NCT number | NCT05022459 |
Other study ID # | STEP Study |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | August 16, 2023 |
Est. completion date | January 2028 |
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.
Status | Recruiting |
Enrollment | 120 |
Est. completion date | January 2028 |
Est. primary completion date | December 2027 |
Accepts healthy volunteers | |
Gender | Male |
Age group | 6 Years to 19 Years |
Eligibility | Inclusion Criteria: 1. 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 2. Male participants with moderate to severe Hemophilia A ( FVIII activity </= 5%) between 6 to 19 years of age without inhibitors are eligible for participation in this study 3. Participants must be on Emicizumab or standard FVIII prophylaxis per institutional/primary hematologist recommendations 4. Participants must be engaging in sports activities with moderate to high risk of bleeding as defined by the NHF- Playing it Safe guidelines (numerical rating >/= 2). 5. Participants must be compliant with completing all standard of care bleed and treatment logs 6. Participant must be willing to keep daily activity logs for the duration of the study. Exclusion Criteria: 1. Participant/parent/LAR unwilling to provide informed consent/assent 2. Unwilling to log or document bleeds and treatment information as per study guidelines 3. Participants with any other bleeding disorders will be excluded 4. Participants on concomittent FVIII replacement and emicizumab for sports participation |
Country | Name | City | State |
---|---|---|---|
United States | Children's Hospital of Michigan | Detroit | Michigan |
Lead Sponsor | Collaborator |
---|---|
Wayne State University | Genentech, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of bleeds | Number of bleeds without trauma, during participation in sports activities | 3 years | |
Secondary | 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. | 3 years | |
Secondary | Change in bone mineral density | Change in bone mineral density as measured by DEXA Scan | 3 years | |
Secondary | 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). | 3 years | |
Secondary | Number of FVIII doses for breakthrough bleeding episodes | Number of doses of factor needed to treat breakthrough bleeding episodes | 3 years | |
Secondary | MRI changes in relevant joints | MRI changes in relevant joints (The joint to be imaged will be determined based on the sport activity; joints most involved in the activity will be imaged). | 3 years |
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