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RECRUITING
NCT05500807
PHASE1

Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A

Sponsor: Bleeding and Clotting Disorders Institute Peoria, Illinois

View on ClinicalTrials.gov

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

DRUG

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