Status:
RECRUITING
Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Lead Sponsor:
Washington Institute for Coagulation
Collaborating Sponsors:
Genentech, Inc.
Conditions:
Hemophilia A
Eligibility:
MALE
16+ years
Brief 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 t...
Detailed Description
Summary: This retrospective/prospective, non-randomized controlled study, whereby patients will be included in 1 of 2 groups depending on their current treatments as outlined below. Patients will be a...
Eligibility Criteria
Inclusion
- 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
- 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)
Key Trial Info
Start Date :
April 4 2019
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
August 1 2026
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT04131036
Start Date
April 4 2019
End Date
August 1 2026
Last Update
September 5 2023
Active Locations (4)
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1
Orthopedic Hemophilia Treatment Center
Los Angeles, California, United States, 90007
2
Hemophilia and Thrombosis Treatment Center, University of California, San Diego
San Diego, California, United States, 92092
3
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States, 78229
4
Washington Center for Bleeding Disorders at Washington Institute for Coagulation
Seattle, Washington, United States, 98101