Hemophilia Clinical Trial
Official title:
Prospective Biomarkers of Bone Metabolism in Hemophilia A
One of the major shortcomings in studying bone disease in hemophilia is the lack of fracture outcome data demonstrating the clinical significance of decreased BMD and altered bone biomarkers in the hemophilia population. This study demonstrates that PwH have an increased risk of fracture compared to the general population and that the issue of bone health will increase in importance as the PwH population ages.
This is a pilot study to determine the impact of factor replacement on bone biomarkers in up
to 20 hemophilia A subjects. Subjects will be recruited over 1 year for the 5-day protocol.
Following a 72-hour washout period, factor levels and bone biomarkers will be followed before
and after 50 units/kg replacement on Day 1 and 20 units/kg replacement on Day 3. Each subject
can serve as their Figure 4. Fracture rates in PwH compared to historic controls.
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