This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
This Phase 3 multicenter, open-label, single-sequence cross-over study will investigate the safety and efficacy of subcutaneous administration of VGA039 as prophylaxis for bleeding in patients with every type of VWD. The study consists of an Observational Period of at least 24 weeks followed by an Active Treatment Period of approximately 49 weeks of VGA039 treatment. Bleeding data and details of treatments used will be collected from each patient during both study periods. The number, duration, location, and types of bleeds experienced, as well as treatments for bleeds, will be recorded in patient diaries. Adverse events will also be monitored and recorded throughout both study periods.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
60
VGA039 SC administered for 49-weeks during the interventional phase following a 24-week observational period.
Arkansas Children's Hospital
Little Rock, Arkansas, United States
RECRUITINGScience 37, Inc. (Virtual Clinical MetaSite)
Morrisville, North Carolina, United States
RECRUITINGHemophilia Center of Western PA
Pittsburgh, Pennsylvania, United States
Incidence of bleeding events [Efficacy]
Time frame: Time Frame: From 7 days after informed consent until 49 weeks after SC study drug initiation
Incidence of treated bleeding events [Efficacy]
Time frame: From 7 days from Informed consent until 49 weeks after SC study drug initiation
Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including hypersensitivity, anaphylaxis, or anaphylactoid reactions
Time frame: From informed consent until 49 weeks after SC study drug initiation
Plasma concentrations of SC doses of VGA039
Time frame: From just prior to the start of study drug administration until 49 weeks after SC study drug initiation
Incidence of Anti-drug antibodies to VGA039
Time frame: From just prior to the start of study drug administration until 49 weeks after SC study drug initiation
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Washington Center for Bleeding Disorders
Seattle, Washington, United States
RECRUITING