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Emicizumab for Severe Von Willebrand Disease and VWD/Hemophilia A

This study is testing if emicizumab (Hemlibra) is a safe and effective way to prevent bleeding in people with severe Von Willebrand Disease (VWD) or VWD combined with hemophilia A. VWD is a common inherited bleeding disorder, and current treatments often require intravenous (IV) infusions. Emicizumab is a bispecific antibody that works by mimicking a clotting factor, and it is given as a subcutaneous (under the skin) injection. This study aims to see if emicizumab can offer a simpler, non-IV option for preventing bleeding. The study plans to enroll about 40 participants, ranging from infants weighing at least 3 kg to adults up to 90 years old. Success will be measured by how well emicizumab prevents bleeding and if it is safe over 18 months.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll about 40 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 18 months to assess the safety and effectiveness of emicizumab.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05500807

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

Recruiting
PHASE1Ages 0–90InterventionalTreatment
Bleeding and Clotting Disorders Institute Peoria, Illinois
~40 participants
Updated 2026-04-03 on ClinicalTrials.gov
What's tested:Emicizumab

At a glance

Recruiting sites
12 of 12 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Emicizumab is efficacious for prophylaxis in severe VWD & concomitant VWD/hemophilia A
Measured over 18 months
+1 more outcome measured

Conditions

Von Willebrand Disease, Type 3
Concomitant VWD and Hemophilia

Explore related trials

Hemophilia Avon Willebrand DiseaseVon Willebrand Factor Human

Where it's being run

12 sites across 9 states
California2
Florida2
Michigan2
Illinois1
Indiana1
Missouri1
Pennsylvania1
Washington1

Study leadership

  • Jonathan C Roberts, MD · PRINCIPAL_INVESTIGATOR · Bleeding and Clotting Disorders Institute

Who to contact

Megan L Woodbury, PhD
[email protected]
309-692-5337 · ext. 174
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Eligibility criteria

Inclusion

Signed informed consent
Age 0 and older (infants weighing ≥3 kg)
ability to comply with protocol in investigators judgement
diagnosis of: severe VWD type 3, or VWD with VWF antigen, activity or collagen binding \</= 20 U/dl or variant VWD confirmed by genetic mutation and VWF ag, activity or CB \< 50 U/dl based on historical medical records of study site.
diagnosis of VWD/hemophilia A defined as VWF:ag, activity or CB \<50 U/dl, and mild moderate or severe hemophilia A(defined by ISTH criteria) based on historical medical records of the study site.
plan to be adherent to emicizumab prophylaxis during the study
Patient's bleeding phenotype necessitating prophylaxis per treating provider recommendations.
Patient on current prophylaxis for VWD or VWD/hemophilia A may enroll if they are currently on a non-emicizumab agent, and if it has been \> 18 months since last off-label dose of emicizumab, and are willing to discontinue current prophylaxis.
For menstruating individuals: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods that result in a failure rate of \< 1% per year during the study period. A menstruating individual is considered to be of childbearing potential if they are post-menarchal, have not reached a postmenopausal state (12 continuous months of amenorrhea with no identified cause other than menopause), and have not undergone surgical sterilization (removal of ovaries and/or uterus).

Exclusion

Patients and/or infants weighing \< 3 kg.
Patients with low VWF or non-severe VWD (ie.not meeting the above criteria)
Other concomitant bleeding disorders including coagulopathy from liver cirrhosis.
Current treatment with emicizumab or emicizumab therapy in the previous 18 months.
Previous (in the past 12 months) or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or current signs of thromboembolic disease
Other conditions (e.g., certain autoimmune diseases, including, but not limited to diseases such as systemic lupus erythematosus, inflammatory bowel disease, and antiphospholipid syndrome) that may increase the risk of bleeding or thrombosis
Patients who are at high risk for thrombotic microangiopathy (TMA; e.g., have a previous medical or family history of TMA), in the investigator's judgment
Would refuse treatment with blood or blood products, if necessary.
Any serious medical condition or abnormality in clinical laboratory tests that, in the investigator's judgment, precludes the patient's safe participation in and completion of the study
Treatment with any of the following:
History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
Pregnant or lactating, or intending to become pregnant during the study
Women of childbearing potential must have a negative serum pregnancy test result within 7 days before Study Day 1
Illicit drug or alcohol abuse within 12 months prior to screening, in the investigator's judgment
Serious infection requiring oral or IV antibiotics within 30 days prior to screening

What this trial measures

  • Emicizumab is efficacious for prophylaxis in severe VWD & concomitant VWD/hemophilia A18 months

    Establish bleed occurrence during treatment evaluated through descriptive statistical analysis to determine proof of principle

  • Emicizumab is safe for prophylaxis in severe VWD & concomitant VWD/Hemophilia A18 months

    Collection of AE's, hypersensitivity reactions, thrombotic events during treatment