Blood

Von Willebrand Disease

Also known as VWD, von Willebrand disorder, bleeding disorder type 1/2/3

Von Willebrand disease is the most common inherited bleeding disorder, caused by quantitative (types 1 and 3) or qualitative (type 2) defects in von Willebrand factor, a multimeric glycoprotein essential for platelet adhesion and factor VII

ORPHA:903 ↗Gene VWFPrevalence Approximately 1 in 100 for all types combined; severe type 3 affects 1 in 1,000,000Onset Childhood to adolescence (often identified at first hemostatic challenge)Autosomal dominant (types 1 and 2) or autosomal recessive (type 3)

33

studies recruiting now

as of 7 Sept 2026

153

studies registered in total

as of 7 Sept 2026

15

countries with a recruiting site

as of 7 Sept 2026

20 Jul 2026

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 33 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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Patient organisations

National Hemophilia FoundationPatient association
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Registry: VWD Connect Patient Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.

About Von Willebrand Disease

Von Willebrand disease is the most common inherited bleeding disorder, caused by quantitative (types 1 and 3) or qualitative (type 2) defects in von Willebrand factor, a multimeric glycoprotein essential for platelet adhesion and factor VIII stabilization. Type 1 is the mildest form with partial deficiency, type 2 encompasses several subtypes with dysfunctional VWF, and type 3 involves near-complete absence of VWF causing severe bleeding similar to hemophilia. Clinical severity correlates poorly with VWF levels alone and requires comprehensive laboratory evaluation.

Common clinical features

Mucocutaneous bleeding including frequent nosebleeds and easy bruisingProlonged bleeding from minor cuts and woundsHeavy menstrual bleeding (menorrhagia) in femalesBleeding after dental procedures or surgeryGastrointestinal bleeding, particularly in type 2A and 2BJoint bleeds and muscle hematomas in severe type 3Postpartum hemorrhageProlonged bleeding after trauma

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

3 approved treatments and 3 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Von Willebrand Factor Human (Von willebrand factor human component of voncento)Approved: Human Coagulation Factor Viii (Hemofil m)Approved: Vonicog Alfa (Veyvondi)
Phase 3Octocog Alfa (Advate)
Phase 3Tranexamic Acid (Cyklo-f heavy period relief)
Phase 2Rondaptivon Pegol

Before you apply

Things trial teams commonly ask about for Von Willebrand Disease. Not eligibility rules; those are set by each study.

  • Specify your VWD subtype (1, 2A, 2B, 2M, 2N, or 3) as trials are often subtype-specific; bring VWF antigen, VWF activity (ristocetin cofactor), and factor VIII levels.
  • Type 2B patients may be excluded from some trials due to thrombocytopenia risk with desmopressin; document any desmopressin (DDAVP) response testing results.
  • Bleeding Assessment Tool (BAT) scores and bleeding history documentation strengthen trial eligibility assessments for novel VWF replacement or gene therapy studies.

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).