Hemophilia A: comparing long-acting FVIII replacement vs non-FVIII therapy in kids

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 is an observational comparative effectiveness study in children with moderate or severe Hemophilia A who are treated with either extended half-life FVIII replacement therapy or a non-FVIII therapy as part of standard care. The researchers will collect joint health data, patient-reported outcomes, and lab results over three years to compare hemostatic potential, annual bleeding rates, and joint health using imaging and the Hemophilia Joint Health Score. The study uses CER methods to analyze differences between the two treatment approaches in real-world practice. Enrollment targets participants who are on prophylaxis with either emicizumab or efanesoctocog alfa for at least two months and are over 6 months old. Key endpoints include bleeding frequency, subclinical joint bleeds, and structural joint changes. This is the first systematic observational comparison of these two conventional therapies in pwHA.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Participants with moderate or severe HA who are on prophylaxis with either weekly, biweekly, or every 4-weeks emicizumab or weekly efanesoctocog alfa for at least 2 months.
* \>6 months of age

Exclusion Criteria:

* Participants with active FVIII inhibitor (\>0.5 BU/mL)
* Presence of an additional bleeding disorder other than hemophilia A

Where it's running

• The University of Texas Southwestern Medical Center
  Dallas, Texas, United States Recruiting

Study contacts

• Study coordinator: Jessica Garcia, MD
• Email: Jessica.Garcia@UTSouthwestern.edu
• Phone: 214-456-7000

Similar studies

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

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

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• Joint health, balance, and quality of life in adults with hemophilia A

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• NXT007 versus emicizumab to prevent bleeds in people with Hemophilia A

  This will test whether NXT007 prevents bleeding better than emicizumab in people aged 12 and older with Hemophilia A, including those with or without factor VIII inhibitors.

  Recruiting at 63 sites Phase 3 Hoffmann-La Roche · Orange, California and 62 other locations · NCT07416604

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.