Comparing Factor VIII and Emicizumab for Preventing Bleeding in Hemophilia A Patients Playing Sports
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
This study tests whether Factor VIII or Emicizumab is better at preventing bleeding in boys aged 6 to 19 with Hemophilia A while they play sports.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 72 (estimated) |
| Ages | 6 Years to 19 Years |
| Sex | All |
| Sponsor | Wayne State University Academic / other |
| Drugs / interventions | Emicizumab |
| Locations | 9 sites (Little Rock, Arkansas and 8 other locations) |
| Trial ID | NCT05022459 on ClinicalTrials.gov |
What this trial studies
This observational study aims to compare the effectiveness of two prophylactic treatments, Factor VIII and Emicizumab, in preventing bleeding episodes in male patients aged 6 to 19 years with moderate to severe Hemophilia A while they engage in sports activities. The study will involve participants who are compliant with treatment logs and are willing to maintain daily activity logs throughout the study period. By assessing the safety and bleeding risk associated with sports participation, the study seeks to identify optimal treatment strategies for these patients.
Who should consider this trial
Good fit: Ideal candidates are male patients aged 6 to 19 years with moderate to severe Hemophilia A who are currently on either Emicizumab or Factor VIII prophylaxis and actively engage in sports.
Not a fit: Patients who are not compliant with treatment logs or who do not participate in sports activities with a moderate to high risk of bleeding may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved safety and quality of life for young patients with Hemophilia A participating in sports.
How similar studies have performed: While the use of Emicizumab has been explored in other studies, this specific comparison with Factor VIII in the context of sports participation is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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. 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 3. Participants must be on Emicizumab or standard FVIII prophylaxis per institutional/primary hematologist recommendations 4. 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). 5. Participant must be willing to keep activity, bleed, and treatment 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. Patients who are pregnant, planning to become pregnant, or breastfeeding should not be enrolled in the study 5. Participants on concomittent FVIII replacement and emicizumab for sports participation
Where this trial is running
Little Rock, Arkansas and 8 other locations
- Arkansas Children's Hospital — Little Rock, Arkansas, United States (Recruiting)
- Stanford University — Palo Alto, California, United States (Recruiting)
- Rady Children's Hospital — San Diego, California, United States (Recruiting)
- Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States (Recruiting)
- Indiana Hemophilia & Thrombosis Center — Indianapolis, Indiana, United States (Recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
- Children's Hospital of Michigan — Detroit, Michigan, United States (Recruiting)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
- Oklahoma Center for Bleeding and Clotting Disorders — Oklahoma City, Oklahoma, United States (Recruiting)
Study contacts
- Principal investigator: Meera Chitlur, MD — Wayne State University
- Study coordinator: Amy Stolinski, MPH
- Email: stepstudy@wayne.edu
- Phone: 313-577-7500
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.