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Blood & Immune

Von Willebrand Disease Clinical Trials and Treatments

Also called vWD, von Willebrand factor deficiency, angiohemophilia

Von Willebrand disease (vWD) is the most common inherited bleeding disorder, affecting 1-3% of the general population with 1 in 10,000 having clinically significant disease. It results from quantitative (Type 1: partial deficiency, Type 3: complete absence) or qualitative (Type 2: dysfunctional) deficiency of von Willebrand factor (vWF), a glycoprotein critical for platelet adhesion and carrier of factor VIII.

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About Von Willebrand Disease

Von Willebrand disease (vWD) is the most common inherited bleeding disorder, affecting 1-3% of the general population with 1 in 10,000 having clinically significant disease. It results from quantitative (Type 1: partial deficiency, Type 3: complete absence) or qualitative (Type 2: dysfunctional) deficiency of von Willebrand factor (vWF), a glycoprotein critical for platelet adhesion and carrier of factor VIII.

Type 1 is autosomal dominant, comprising approximately 75% of cases. Type 3 is autosomal recessive with complete vWF absence and most severe phenotype. Type 2 encompasses multiple dysfunctional variants with variable inheritance patterns. Pathophysiology involves impaired platelet adhesion to damaged endothelium and reduced factor VIII levels.

Clinical manifestations include mucosal bleeding (epistaxis, gingival bleeding, GI bleeding), heavy menstrual bleeding (particularly symptomatic in women), and excessive bleeding with trauma or surgery. Bleeding severity varies within the same type and even within affected families. Type 2 variants show markedly variable presentation. Type 3 patients have complete absence of functional vWF and factor VIII deficiency approaching hemophilia severity. Diagnosis requires vWF activity and antigen level measurement, factor VIII activity assay, and bleeding phenotype assessment.

Common Symptoms of Von Willebrand Disease

Recognizing the signs of Von Willebrand Disease early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Easy bruising and mucosal bleeding
  • Heavy menstrual bleeding in women
  • Nosebleeds
  • Gum bleeding
  • Prolonged bleeding after trauma or surgery
  • Gastrointestinal bleeding in severe forms

Who Von Willebrand Disease Affects

Affects males and females equally in Types 1 and 3. Women disproportionately affected by bleeding symptoms due to menstrual blood loss and pregnancy-related complications. All ethnic backgrounds affected with similar prevalence. Type 1 autosomal dominant; Types 2 and 3 variable inheritance with Type 3 autosomal recessive.

Geographic variation minimal though some ethnic populations show higher incidence. African American, Caucasian, and Hispanic populations show prevalence approaching 1-3%. Asian populations may have lower reported prevalence. Type 2 variants show dramatic geographic variation.

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Help Paying for Von Willebrand Disease Treatment

Charity funds and drugmaker programs for Von Willebrand Disease, checked at the source. Pick your insurance to see what fits.

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Charity funds
  • From a charity · Hemophilia Federation of America
    Helping Hands Program fund
    Open

    Pays for: Emergency living expenses ($250 prepaid card, once per year; referral through HTC or member organization; does not cover medical bills, copays or premiums), up to $250 per year.

    The foundation says: “The Helping Hands program is now open on a limited basis for 2026. Due to funding constraints, a maximum of 20 requests will be approved…”
Status as each foundation showed it on September 28, 2026.

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Side Effect Explorer

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

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Trusted Von Willebrand Disease Resources

Reputable organizations and medical references for learning more about Von Willebrand Disease, including disease registries, foundation resources, and clinical guidelines.

Active Clinical Trials for Von Willebrand Disease

Use this Von Willebrand Disease clinical trial finder to see the 19 studies recruiting patients and 1 opening soon in the United States and worldwide, with eligibility criteria in plain English. These studies play a critical role in advancing care for blood & immune conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

TrialsSite mapPipeline timeline

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

35 active trials worldwide
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RECRUITINGPHASE2Recently updatedNCT07575308

HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB

Intervention: ALN-6400

Sponsor: Alnylam Pharmaceuticals

The purpose of this study is to evaluate the safety, tolerability, efficacy, and pharmacodynamics (PD) of multiple doses of ALN-6400 in adult and adolescent patients with VWD and HMB

Ages 16 Years – 45 Years11 locations
Started Jul 2026Updated 2 weeks agoEst. Jun 2027 (~9 months)
RECRUITINGPHASE3Recently updatedNCT07115004

Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)

Intervention: VGA039

Sponsor: Vega Therapeutics, Inc

This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)

Ages 12 Years – 75 Years32 locations
Started Oct 2025Updated 2 weeks agoEst. Oct 2028 (~2 years)
RECRUITINGRecently updatedNCT07418099

External Multicenter Validation of the APTTO Model for Prolonged APTT Using Clot Waveform Analysis

Intervention: Clot waveform analysis-based risk stratification (APTTO models)

Sponsor: Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz

Prolonged activated partial thromboplastin time (APTT) is a frequent laboratory finding that may reflect a broad spectrum of underlying conditions, ranging from benign laboratory abnormalities to clinically relevant hemostatic disorders. Clot waveform analysis (CWA), automaticall...

Ages not specified16 locations
Started Jan 2026Updated 3 weeks agoEst. Mar 2027 (~5 months)
RECRUITINGPHASE3Recently updatedNCT05582993

A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)

Intervention: Vonicog Alfa, ADVATE

Sponsor: Takeda

The main aim of the study is to evaluate the effectiveness of prophylaxis with vonicog alfa (recombinant von Willebrand factor [rVWF]) in children. This study will enroll those participants who have been previously treated with VWF product or with a plasma-derived VWF (pdVWF) pro...

Ages up to 17 Years23 locations
Started Nov 2024Updated 3 weeks agoEst. Apr 2030 (~3y 6m)
RECRUITINGPHASE3Recently updatedNCT06998524

A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease

Intervention: Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates, von Willebrand Factor (VWF) and Factor VIII (FVIII) Concentrates, Bypassing Agents

Sponsor: Hoffmann-La Roche

This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von Willebrand disease (VWD). Partic...

Ages 1 Month+29 locations
Started Jun 2025Updated 3 weeks agoEst. Apr 2027 (~6 months)
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Active trial locations86 cities in the US
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Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
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Related Blood & Immune Conditions

Other rare diseases in the blood & immune category. Patients with Von Willebrand Disease may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Von Willebrand Disease Treatments

6 pharmaceutical companies have Von Willebrand Disease in their rare disease portfolio

Frequently Asked Questions About Von Willebrand Disease