The VIVID study is structured in a master protocol format comprised of multiple parts that evaluate intravenous (IV) and subcutaneous (SC) VGA039 in healthy volunteers and subjects with von Willebrand Disease (VWD) and other bleeding disorders.
This first-in-human study consists of 5 parts based on the subject population. Part 1 is a randomized, double-blind, placebo-controlled, single-ascending dose (SAD) evaluation of IV or SC VGA039 or placebo in up to 8 cohorts in healthy volunteers. Part 2 is an open-label, SAD of SC or IV VGA039 in up to 8 cohorts in subjects diagnosed with VWD. All participants will be enrolled, treated, and followed up for 15 weeks (IV SAD) or 8 weeks(SC SAD). Part 3 is an open-label, Phase 1b study of SC multiple doses (MD) of VGA039 in up to 4 cohorts. Part 4 is an open-label, Phase 2 study of SC single, surgical prophylaxis (SP) doses of VGA039 administered prior to a minor surgical procedure in subjects diagnosed with VWD in up to 2 cohorts. Part 5 is an open-label extension (OLE) study of SC MD of VGA039 in eligible subjects diagnosed with VWD who have previously participated in a VGA039 interventional trial.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
116
Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]
Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including dose-limiting toxicities (DLTs).
Time frame: From start of study drug administration until 15 or 8 weeks after IV or SC study drug administration, respectively
Plasma Concentrations of single IV and SC doses of VGA039
Time frame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
Pharmacodynamics of single IV and SC doses of VGA039
Time frame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
Incidence of Anti-drug antibodies to VGA039
Time frame: From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
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