RecruitingPhase 1NCT05500807

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

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


Sponsor

Bleeding and Clotting Disorders Institute Peoria, Illinois

Enrollment

40 participants

Start Date

Nov 1, 2022

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 0 YearsMax Age: 90 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Emicizumab for people with concomitant vwd and hemophilia and von willebrand disease, type 3. The study is currently recruiting participants at 12 locations. People eligible for this study include aged 0 Years to 90 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGEmicizumab

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

View Full Details on ClinicalTrials.gov

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NCT05500807


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