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Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
Sponsor: Bleeding and Clotting Disorders Institute Peoria, Illinois
Summary
Von Willebrand Disease (VWD) is the most common inherited bleeding disorder affecting up to 0.1% of the population, is usually characterized by mucocutaneous bleeding, HMB, surgical bleeding or other hemostatic challenges. Severe bleeding events require VWF concentrates administered solely through intravenous access. Emicizumab (Hemlibra) is a monoclonal bispecific antibody developed to bind activated FIX and FX and mimic FVIII cofactor functionality. Hemlibra is administered via subcutaneous injection rather than intravenous infusion. The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWD/hemophilia patients.
Official title: Emicizumab for Severe VON Willebrand Disease (VWD) and VWD/Hemophilia A
Key Details
Gender
All
Age Range
0 Years - 90 Years
Study Type
INTERVENTIONAL
Enrollment
40
Start Date
2022-11-01
Completion Date
2028-02
Last Updated
2026-04-03
Healthy Volunteers
No
Interventions
Emicizumab
Subcutaneous injection of emicizumab for prophylaxis
Locations (12)
The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD)
Orange, California, United States
Stanford University: Stanford Children's Health
Redwood City, California, United States
University of Miami - Miller School of Medicine
Coral Gables, Florida, United States
St. Joseph's Children's Hospital - Center for Bleeding and Clotting Disorders
Tampa, Florida, United States
Bleeding and Clotting Disorders Institute (BCDI)
Peoria, Illinois, United States
Innovative Hematology, Inc. (IHI)
Indianapolis, Indiana, United States
University of Michigan Medical School
Ann Arbor, Michigan, United States
Central Michigan University: Children's Hospital of Michigan
Mount Pleasant, Michigan, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
Penn State College of Medicine
Hershey, Pennsylvania, United States
Washington Center for Bleeding Disorders
Seattle, Washington, United States
UW Health Comprehensive Program for Bleeding Disorders
Madison, Wisconsin, United States