Study of VGA039 in Healthy Volunteers and Patients with Von Willebrand Disease (VIVID)
- Registration Number
- NCT05776069
- Lead Sponsor
- Vega Therapeutics, Inc
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 116
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part 1 VGA039 Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined Part 1 Placebo Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined Part 2 VGA039 Cohorts A-H IV or SC VGA039 dose to be determined Part 3 VGA039 Cohorts MD-1 to MD-4, SC VGA039 multiple doses, dose to be determined Part 4 VGA039 Cohorts of VGA039 single dose for surgical prophylaxis
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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
Trial Locations
- Locations (24)
Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory
🇺🇸Atlanta, Georgia, United States
Washington Center for Bleeding Disorders
🇺🇸Seattle, Washington, United States
Royal Brisbane & Women's Hospital, Queensland Haemophilia Centre
🇦🇺Herston, Queenland, Australia
Queen Elizabeth Hospital Birmingham
🇬🇧Birmingham, Edgbaston, United Kingdom
University Hospital Southampton NHS Foundation Trust
🇬🇧Southampton, Hampshire, United Kingdom
Orthopedic Institute for Children (UCLA)
🇺🇸Los Angeles, California, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
University of Colorado School of Medicine
🇺🇸Aurora, Colorado, United States
Hemophilia Center of Western PA
🇺🇸Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
University of Texas Southwestern
🇺🇸Dallas, Texas, United States
Versiti Comprehensive Center for Bleeding Disorders
🇺🇸Milwaukee, Wisconsin, United States
Medical University of Vienna
🇦🇹Vienna, Austria
Hemocentro Unicamp
🇧🇷Campinas, Sao Paulo, Brazil
Centro de Hemoterapia e Hematologia do Rio de Janeiro HEMORIO
🇧🇷Rio De Janeiro, Brazil
Hospital das Clinicas - USP Endereco
🇧🇷São Paulo, Brazil
Hamilton Health Sciences Corporation
🇨🇦Hamilton, Ontario, Canada
Queens University
🇨🇦Kingston, Ontario, Canada
St. Michaels Hospital
🇨🇦Toronto, Ontario, Canada
K J Somaiya Super Speciality Hospital & Research Centre
🇮🇳Sion, Mumbai, India
Charlotte Maxeke Johannesburg Academic Hospital
🇿🇦Johannesburg, South Africa
Royal London Hospital, Clinical Haematology Research
🇬🇧Whitechapel, London, United Kingdom
Royal Free Hospital
🇬🇧London, United Kingdom
Imperial College Healthcare NHS Trust- Queen Charlotte's & Chelsea Hospital
🇬🇧London, United Kingdom