What type of study is this?

This is a Human Clinical Trial study (also classified as: Phase 3, Pre-Registered Trial).

What question did this study set out to answer?

To evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of VGA039 in patients with von Willebrand disease.

December 8, 2025BloodOpen Access

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Open-label trial shows subcutaneous VGA039 significantly improves bleeding control in von Willebrand disease, suggesting its efficacy.

To evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of VGA039 in patients with von Willebrand disease.
Open-label phase 1/2 study conducted in adolescents and adults with VWD
Participants received multiple subcutaneous doses of VGA039 over 120 days
Weekly diaries recorded information on bleeding and treatments
No drug-related adverse events were reported, with only mild headache in one patient
Substantial 74% reduction in bleeding rates compared to historical prophylaxis
All patients maintained target concentrations of VGA039 without significant D-dimer elevations

Wheeler et al. (2025) studied this question.

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