Comparing Factor VIII and Emicizumab for Preventing Bleeding in Hemophilia A Patients Playing Sports

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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
• Rush University Children's Hospital
  Chicago, Illinois, United States Recruiting
• Indiana Hemophilia & Thrombosis Center
  Indianapolis, Indiana, 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

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• SR604: a new treatment tested in adults with Hemophilia A/B or congenital Factor VII deficiency

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• Bone health in severe hemophilia A: emicizumab vs efanesoctocog alfa

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.