Clinical Trial

A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)

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Summary
This study will compare bone mineral density in patients with severe hemophilia A receiving prophylaxis with emicizumab or efanesoctocog alfa. Participants will undergo assessments of bone mineral density, bone remodeling biomarkers, thrombin generation, plasmin generation, and joint health over a five-year period. The study aims to evaluate whether differences in prophylactic therapy are associated with differences in bone health outcomes.
Protocol Amendment History 1 change
critical Primary endpoint(s) modified 2026-06-05
Trial Details
NCT Number NCT07582276
Lead Sponsor Arkansas Children's Hospital Research Institute
Collaborators: Genzyme, a Sanofi Company
Conditions Hemophilia a
Enrollment 50 participants
Start Date 2026-07-01
Primary Completion 2031-08-01 (estimated)
Study Completion 2032-08-01 (estimated)
Updated on ClinicalTrials.gov 2026-06-04