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NCT05776069PHASE1, PHASE2RecruitingAccepts Healthy Volunteers

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

Vega Therapeutics, Inc

Start Date

3/16/2023

Completion Date

12/1/2029

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.

Eligibility Criteria

Age Range: 12 years to 60 years

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.

Interventions

DRUG

VGA039

OTHER

Placebo

DRUG

VGA039

Interested in This Trial?

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Conditions

Von Willebrand Diseases

Locations

Orthopedic Institute for Children (UCLA)

Los Angeles, California 90007

United States

UC Davis Medical Center

Sacramento, California 95817

United States

University of Colorado School of Medicine

Aurora, Colorado 80045

United States

Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory

Atlanta, Georgia 30308

United States

Science 37, Inc.

Morrisville, North Carolina 27560

United States

Hemophilia Center of Western PA

Pittsburgh, Pennsylvania 15213

United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232

United States

University of Texas Southwestern

Dallas, Texas 75235

United States

Washington Center for Bleeding Disorders

Seattle, Washington 98101

United States

Versiti Comprehensive Center for Bleeding Disorders

Milwaukee, Wisconsin 53233

United States

Royal Brisbane & Women's Hospital, Queensland Haemophilia Centre

Herston, Queenland 4029

Australia

Medical University of Vienna

Vienna, State of Vienna

Austria

Centro de Hemoterapia e Hematologia do Rio de Janeiro HEMORIO

Rio de Janeiro, Rio de Janeiro 20211-030

Brazil

Hemocentro Unicamp

Campinas, São Paulo 13083-878

Brazil

Hospital das Clinicas - USP Endereco

São Paulo, São Paulo 05403-010

Brazil

Hamilton Health Sciences Corporation

Hamilton, Ontario L8N 3Z5

Canada

Queens University

Kingston, Ontario K7L 3N6

Canada

St. Michaels Hospital

Toronto, Ontario M5B 1W8

Canada

K J Somaiya Super Speciality Hospital & Research Centre

Sion, Mumbai 400022

India

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg,

South Africa

Queen Elizabeth Hospital Birmingham

Birmingham, Edgbaston B15 2TT

United Kingdom

University Hospital Southampton NHS Foundation Trust

Southampton, Hampshire SO16 6YD.

United Kingdom

Royal Free Hospital

London, London NW3 2QG

United Kingdom

Royal London Hospital, Clinical Haematology Research

Whitechapel, London E12ES

United Kingdom

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

London,

United Kingdom