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

NCT05776069 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: VGA039-CP001
• Study Type: interventional
• Target: 116
• Locations: 7 countries
• Sites: 24

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.

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

Outcome Measures

Primary Outcomes (1)

• 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).

  From start of study drug administration until 15 or 8 weeks after IV or SC study drug administration, respectively

Secondary Outcomes (3)

• 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

Study Arms (5)

Part 1

PLACEBO COMPARATOR

Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined

Drug: VGA039; Other: Placebo

Part 2

EXPERIMENTAL

Cohorts A-H IV or SC VGA039 dose to be determined

Drug: VGA039

Part 3

EXPERIMENTAL

Cohorts MD-1 to MD-4, SC VGA039 multiple doses, dose to be determined

Drug: VGA039

Part 4

EXPERIMENTAL

Cohorts of VGA039 single dose for surgical prophylaxis

Drug: VGA039

Part 5

EXPERIMENTAL

Multiple doses of VGA039 in open label extension

Interventions

VGA039 DRUG

Single doses of VGA039

Part 1; Part 2; Part 4

Placebo OTHER

Single doses of Placebo

Part 1

Eligibility Criteria

• Age: 12 Years - 60 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• 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.
• 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.
• Baseline FVIII activity \> 150 IU/dL.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Vega Therapeutics, Inc: lead

Study Sites (25)

Orthopedic Institute for Children (UCLA)

Los Angeles, California, 90007, United States

RECRUITING

UC Davis Medical Center

Sacramento, California, 95817, United States

RECRUITING

University of Colorado School of Medicine

Aurora, Colorado, 80045, United States

RECRUITING

Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory

Atlanta, Georgia, 30308, United States

RECRUITING

Science 37, Inc.

Morrisville, North Carolina, 27560, United States

RECRUITING

Hemophilia Center of Western PA

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

University of Texas Southwestern

Dallas, Texas, 75235, United States

RECRUITING

Washington Center for Bleeding Disorders

Seattle, Washington, 98101, United States

RECRUITING

Versiti Comprehensive Center for Bleeding Disorders

Milwaukee, Wisconsin, 53233, United States

RECRUITING

Royal Brisbane & Women's Hospital, Queensland Haemophilia Centre

Herston, Queenland, 4029, Australia

RECRUITING

Medical University of Vienna

Vienna, Vienna, Austria

COMPLETED

Centro de Hemoterapia e Hematologia do Rio de Janeiro HEMORIO

Rio de Janeiro, Rio de Janeiro, 20211-030, Brazil

RECRUITING

Hemocentro Unicamp

Campinas, São Paulo, 13083-878, Brazil

RECRUITING

Hospital das Clinicas - USP Endereco

São Paulo, São Paulo, 05403-010, Brazil

RECRUITING

Hamilton Health Sciences Corporation

Hamilton, Ontario, L8N 3Z5, Canada

RECRUITING

Queens University

Kingston, Ontario, K7L 3N6, Canada

RECRUITING

St. Michaels Hospital

Toronto, Ontario, M5B 1W8, Canada

RECRUITING

K J Somaiya Super Speciality Hospital & Research Centre

Sion, Mumbai, 400022, India

RECRUITING

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg, South Africa

RECRUITING

Queen Elizabeth Hospital Birmingham

Birmingham, Edgbaston, B15 2TT, United Kingdom

RECRUITING

University Hospital Southampton NHS Foundation Trust

Southampton, Hampshire, SO16 6YD., United Kingdom

RECRUITING

Royal Free Hospital

London, London, NW3 2QG, United Kingdom

RECRUITING

Royal London Hospital, Clinical Haematology Research

Whitechapel, London, E12ES, United Kingdom

RECRUITING

Imperial College Healthcare NHS Trust- Queen Charlotte's & Chelsea Hospital

London, United Kingdom

RECRUITING

MeSH Terms

Conditions

von Willebrand Diseases; Hemostatic Disorders; Blood Coagulation Disorders

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Vascular Diseases; Cardiovascular Diseases

Central Study Contacts

Clinical Trials

CONTACT

650-466-8041; medinfo@star-therapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 8, 2023
• First Posted: March 20, 2023
• Study Start: March 16, 2023
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: August 26, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

CA (3); AU (1); US (10); GB (5); BR (3); ZA (1); IN (1)