Actively Recruiting

Phase 1
Age: 0Years - 90Years
All Genders
ID05500807

Emicizumab for Severe Von Willebrand Disease and VWD with Hemophilia A

Led by Bleeding and Clotting Disorders Institute Peoria, Illinois · Updated on 2026-04-03

40

Participants Needed

12

Research Sites

35 weeks

Total Duration

On this page

Sponsors

B

Bleeding and Clotting Disorders Institute Peoria, Illinois

Lead Sponsor

G

Genentech, Inc.

Collaborating Sponsor

AI-Summary

What this Trial Is About

Von Willebrand Disease (VWD) is the most common inherited bleeding disorder, affecting up to 0.1% of people. It often causes mucous membrane bleeding, heavy menstrual bleeding, surgical bleeding, or other bleeding challenges. Severe cases require treatment with von Willebrand factor (VWF) concentrates given intravenously. This study is evaluating emicizumab, a monoclonal antibody given by subcutaneous injection, as a potential prophylactic treatment for severe VWD and patients with both VWD and hemophilia A. Participants will receive emicizumab starting with a loading dose of 3 mg/kg once weekly for 4 weeks, followed by a maintenance dose of 1.5 mg/kg once weekly for one year. Dose escalation to 3 mg/kg weekly may occur after 24 weeks if bleeding control is insufficient. Breakthrough bleeding can be treated with usual therapies such as antifibrinolytics or VWF/FVIII concentrates. The study is open-label and will collect treatment records and bleeding logs throughout. Participants will be monitored for efficacy and safety over 18 months, including patient-reported outcomes on quality of life, physical, emotional, social, and general symptoms. Data on annualized bleed rates and treatment burden compared to previous therapies will be collected. Safety assessments and adherence to emicizumab prophylaxis will be tracked as well. This includes a one-year retrospective review of bleeding history before starting treatment.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 0Years - 90Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Signed informed consent
  • Age 0 years and older (infants weighing at least 3 kg)
  • Ability to comply with study protocol as judged by investigator
  • Diagnosis of severe VWD type 3, or VWD with VWF antigen, activity, or collagen binding ≤ 20 U/dl, or variant VWD with VWF antigen, activity, or collagen binding < 50 U/dl confirmed by medical records
  • Diagnosis of VWD/hemophilia A with VWF:ag, activity or collagen binding <50 U/dl and mild, moderate, or severe hemophilia A per ISTH criteria
  • Plan to adhere to emicizumab prophylaxis during the study
  • Bleeding phenotype requiring prophylaxis per treating provider
  • If currently on prophylaxis (non-emicizumab), must have discontinued emicizumab for at least 18 months and willing to stop current prophylaxis
  • For menstruating individuals: agreement to remain abstinent or use highly effective contraception with failure rate <1% per year during study
Not Eligible

You will not qualify if you...

  • Weight less than 3 kg
  • Patients with low VWF or non-severe VWD
  • Other bleeding disorders such as coagulopathy from liver cirrhosis
  • Current or recent (within 18 months) treatment with emicizumab
  • Recent or current thromboembolic disease treatment or signs
  • Certain autoimmune diseases increasing bleeding or thrombosis risk
  • High risk for thrombotic microangiopathy as judged by investigator
  • Refusal of treatment with blood or blood products if needed
  • Serious medical conditions or lab abnormalities precluding safe participation
  • Recent use of investigational drugs for hemophilic bleeding or other conditions
  • History of hypersensitivity to monoclonal antibodies or emicizumab components
  • Pregnant, lactating, or intending to become pregnant during study
  • Women of childbearing potential without negative pregnancy test within 7 days before study
  • Illicit drug or alcohol abuse within 12 months prior to screening
  • Serious infection requiring antibiotics within 30 days prior to screening

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Approximately 1 year

Participants receive subcutaneous injections of emicizumab as a prophylactic treatment for severe Von Willebrand Disease or VWD with Hemophilia A. The treatment starts with a weekly loading dose for 4 weeks, followed by weekly maintenance doses for up to 1 year. Dose adjustments may occur after 24 weeks if needed.

Weekly visits during the first 4 weeks, then weekly visits for maintenance dosing

Trial Site Locations

Total: 12 locations

1

The Center for Comprehensive Care and Diagnosis of Inherited Blood Disorders (CIBD)

Orange, California, United States, 92868

Actively Recruiting

2

Stanford University: Stanford Children's Health

Redwood City, California, United States, 94063

Actively Recruiting

3

University of Miami - Miller School of Medicine

Coral Gables, Florida, United States, 33146

Actively Recruiting

4

St. Joseph's Children's Hospital - Center for Bleeding and Clotting Disorders

Tampa, Florida, United States, 33607

Actively Recruiting

5

Bleeding and Clotting Disorders Institute (BCDI)

Peoria, Illinois, United States, 61614

Actively Recruiting

6

Innovative Hematology, Inc. (IHI)

Indianapolis, Indiana, United States, 46260

Actively Recruiting

7

University of Michigan Medical School

Ann Arbor, Michigan, United States, 48109

Actively Recruiting

8

Central Michigan University: Children's Hospital of Michigan

Mount Pleasant, Michigan, United States, 48859

Actively Recruiting

9

Children's Mercy Hospital

Kansas City, Missouri, United States, 64108

Actively Recruiting

10

Penn State College of Medicine

Hershey, Pennsylvania, United States, 17033

Actively Recruiting

11

Washington Center for Bleeding Disorders

Seattle, Washington, United States, 98101

Actively Recruiting

12

UW Health Comprehensive Program for Bleeding Disorders

Madison, Wisconsin, United States, 53792

Actively Recruiting

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Research Team

M

Megan L Woodbury, PhD

D

Dayna Lenski, BS

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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Published Research Related To This Trial

Combined haemophilia A and type I von Willebrand's disease: a family study including an evaluation of the effects of DDAVP infusion.

A Casonato, E Pontara, M Boscaro...

https://pubmed.ncbi.nlm.nih.gov/8339998

A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model.

Takehisa Kitazawa, Tomoyuki Igawa, Zenjiro Sampei...

https://pubmed.ncbi.nlm.nih.gov/23023498