Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease (VIVID)

Conditions

Von Willebrand Diseases

Keywords

Von Willebrand Disorder, VWD, Star Therapeutics, Vega Therapeutics, VIVID, VIVID-1, VIVID-2, VIVID-3, VIVID-5, Bleeding disorders, VGA039, Blood coagulation disorders

Brief summary

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.

Detailed description

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.

Allison P. Wheeler, Shrinath Kshirsagar, Adam Giermasz, Johnny Mahlangu, Gabriela G Yamaguti-Hayakawa et al. (17 authors)

Blood2025-11-03

10.1182/blood-2025-308

VGA039 as a protein S-targeted hemostatic promoting monoclonal antibody, promotes in-vitro thrombin generation in plasma samples from subjects across a broad range of bleeding disorders, including von Willebrand disease, hemophilia A, hemophilia B and hemophilia C

Alina T. He, Paul Sims, Joanne Chan, Christopher Horvath, Benjamin Kim et al. (11 authors)

Blood2025-11-03

10.1182/blood-2025-1277

A Phase Ia Study of VGA039, a Protein S-Targeting Monoclonal Antibody, in Individuals with Von Willebrand Disease Demonstrates Concentration-Dependent Increases in Thrombin Generation for Reducing Bleeding

Carolyn M. Millar, Priyanka Raheja, Allison P. Wheeler, Shrinath Kshirsagar, Christian Schoergenhofer et al. (14 authors)

Blood2024-11-054 citations

10.1182/blood-2024-208020

Interventions

DRUGVGA039

Single doses of VGA039

OTHERPlacebo

Single doses of Placebo

Study design

Allocation

RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

ALL

Age

12 Years to 60 Years

Healthy volunteers

Yes

Inclusion criteria

Key Inclusion Criteria (All Subjects) * Subjects, 18 to 60 years of age, inclusive for Parts 1 and 2 * Subjects, 12 to 60 years of age, inclusive for Parts 3 and 5 * No clinically significant laboratory, ECG, or vital signs results. Additional Key Inclusion Criteria (for Subjects in Part 1 Only) • Body mass index of 18-32 kg/m2 Additional Key Inclusion Criteria (for Subjects in Part 2 Only) * Subjects with VWD who are symptomatic, defined as having a history of bleeding or bruising. * Hemoglobin level ≥ 8 g/dL and platelet count ≥ 150 × 109/L at Screening. Exclusion Key Criteria (All Subjects) * Use of hormonal contraceptives within 56 days prior to administration of the study drug. * Subjects with detection of FV Leiden or Prothrombin G20210A mutation, protein C or S deficiency, antithrombin deficiency, or antiphospholipid antibody syndrome at Screening. * Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation. * History of arterial or venous thrombosis, including superficial thrombophlebitis, or embolism. * Evidence of renal, hepatic, central nervous system, respiratory, cardiovascular disease, cerebrovascular disease, peripheral vascular disease, or metabolic dysfunction. Additional Key Exclusion Criterion (Subjects in Part 1 Only) • Baseline FVIII activity \> 150 IU/dL. Additional Key

Exclusion criteria

(Subjects in Parts 2, 3, 4 and 5 Only) * Baseline FVIII activity \> 50 IU/dL. * Any acute, clinically significant bleeding event requiring surgical or procedural intervention within 7 days prior to receiving study drug.

Design outcomes

Primary

Measure	Time frame	Description
Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]	From start of study drug administration until 15 or 8 weeks after IV or SC study drug administration, respectively	Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including dose-limiting toxicities (DLTs).

Secondary

Measure	Time frame
Plasma Concentrations of single IV and SC doses of VGA039	From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
Pharmacodynamics of single IV and SC doses of VGA039	From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively
Incidence of Anti-drug antibodies to VGA039	From baseline until 15 or 8 weeks after IV or SC study drug administration, respectively

Countries

Australia, Austria, Brazil, Canada, India, South Africa, United Kingdom, United States

Contacts

Primary ContactClinical Trials

medinfo@star-therapeutics.com650-466-8041

Outcome results

None listed