Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Washington Institute for Coagulation
40 participants
Apr 4, 2019
OBSERVATIONAL
Conditions
Summary
The investigators propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A. Per current standard of care, most patients are on prophylactic FVIII replacement therapy intravenously several times weekly with a goal of keeping the trough \>1% FVIII. Recent phase 3 data suggest superior bleed protection with emicizumab prophylaxis every 1-2 weeks. It is the purpose of this study to longitudinally assess joint health and bone density over 3 years and to compare the effect of routine factor VIII prophylaxis with emicizumab prophylaxis.
Eligibility
Inclusion Criteria8
- Male gender
- Severe hemophilia A (factor VIII < 1%)
- Age ≥ 16 year
- Either on prophylaxis with factor VIII or emicizumab with the intention to stay on the current regimen for the next 3 years
- Willing and able to give written informed consent/assent
- Willing to undergo MSKUS, DEXA scan +/- collection of blood sampling for repository biomarkers
- Willing to come in for baseline and 3 yearly visits
- Willing to answer phone survey for bleeding and safety every 3 months
Exclusion Criteria5
- Current FVIII inhibitor of > 0.6 BU
- Unable to take FVIII replacement
- Other known bleeding disorder
- Other rheumatologic disorder affecting joints
- Other known neuromotor defect (making physical exam difficult)
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Interventions
We propose to study longitudinal joint and bone density changes in patients with severe Hemophilia A.
Locations(4)
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NCT04131036