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

NCT ID: NCT05776069

Last Updated: 2025-08-26

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

116 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-03-16

Study Completion Date

2029-12-31

Brief Summary

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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

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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.

Conditions

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Von Willebrand Diseases

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Part 1

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

Group Type PLACEBO_COMPARATOR

VGA039

Intervention Type DRUG

Single doses of VGA039

Placebo

Intervention Type OTHER

Single doses of Placebo

Part 2

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

Group Type EXPERIMENTAL

VGA039

Intervention Type DRUG

Single doses of VGA039

Part 3

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

Group Type EXPERIMENTAL

VGA039

Intervention Type DRUG

Multiple doses of VGA039

Part 4

Cohorts of VGA039 single dose for surgical prophylaxis

Group Type EXPERIMENTAL

VGA039

Intervention Type DRUG

Single doses of VGA039

Part 5

Multiple doses of VGA039 in open label extension

Group Type EXPERIMENTAL

No interventions assigned to this group

Interventions

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VGA039

Single doses of VGA039

Intervention Type DRUG

Placebo

Single doses of Placebo

Intervention Type OTHER

VGA039

Multiple doses of VGA039

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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.

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.

Exclusion Criteria

* 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.
Minimum Eligible Age

12 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Vega Therapeutics, Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Orthopedic Institute for Children (UCLA)

Los Angeles, California, United States

Site Status RECRUITING

UC Davis Medical Center

Sacramento, California, United States

Site Status RECRUITING

University of Colorado School of Medicine

Aurora, Colorado, United States

Site Status RECRUITING

Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory

Atlanta, Georgia, United States

Site Status RECRUITING

Science 37, Inc.

Morrisville, North Carolina, United States

Site Status RECRUITING

Hemophilia Center of Western PA

Pittsburgh, Pennsylvania, United States

Site Status RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status RECRUITING

University of Texas Southwestern

Dallas, Texas, United States

Site Status RECRUITING

Washington Center for Bleeding Disorders

Seattle, Washington, United States

Site Status RECRUITING

Versiti Comprehensive Center for Bleeding Disorders

Milwaukee, Wisconsin, United States

Site Status RECRUITING

Royal Brisbane & Women's Hospital, Queensland Haemophilia Centre

Herston, Queenland, Australia

Site Status RECRUITING

Medical University of Vienna

Vienna, Vienna, Austria

Site Status COMPLETED

Centro de Hemoterapia e Hematologia do Rio de Janeiro HEMORIO

Rio de Janeiro, Rio de Janeiro, Brazil

Site Status RECRUITING

Hemocentro Unicamp

Campinas, São Paulo, Brazil

Site Status RECRUITING

Hospital das Clinicas - USP Endereco

São Paulo, São Paulo, Brazil

Site Status RECRUITING

Hamilton Health Sciences Corporation

Hamilton, Ontario, Canada

Site Status RECRUITING

Queens University

Kingston, Ontario, Canada

Site Status RECRUITING

St. Michaels Hospital

Toronto, Ontario, Canada

Site Status RECRUITING

K J Somaiya Super Speciality Hospital & Research Centre

Sion, Mumbai, India

Site Status RECRUITING

Charlotte Maxeke Johannesburg Academic Hospital

Johannesburg, , South Africa

Site Status RECRUITING

Queen Elizabeth Hospital Birmingham

Birmingham, Edgbaston, United Kingdom

Site Status RECRUITING

University Hospital Southampton NHS Foundation Trust

Southampton, Hampshire, United Kingdom

Site Status RECRUITING

Royal Free Hospital

London, London, United Kingdom

Site Status RECRUITING

Royal London Hospital, Clinical Haematology Research

Whitechapel, London, United Kingdom

Site Status RECRUITING

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

London, , United Kingdom

Site Status RECRUITING

Countries

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United States Australia Austria Brazil Canada India South Africa United Kingdom

Central Contacts

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Clinical Trials

Role: CONTACT

650-466-8041

Facility Contacts

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Science 37 Recruitment Team

Role: primary

984-377-3737

Other Identifiers

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VGA039-CP001

Identifier Type: -

Identifier Source: org_study_id

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