Phase 3 study of VGA039 prophylaxis in adolescent and adult patients with von Willebrand Disease
Research type
Research Study
Full title
A Prospective, Multicenter, Open-label, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of Prophylactic VGA039 in Adolescent and Adult Patients with von Willebrand Disease (VIVID-6)
IRAS ID
1012495
Contact name
Benjamin Kim
Contact email
Sponsor organisation
Vega Therapeutics Inc.
Eudract number
2025-522056-10
Clinicaltrials.gov Identifier
Research summary
Rationale: Von Willebrand disease (VWD) is a common bleeding disorder caused by a problem with von Willebrand Factor (VWF), a protein that helps blood clot. Symptoms include easy bruising, frequent nosebleeds, excessive bleeding after injury or surgery, and heavy menstrual periods. VWD affects about 1 in 100 people.
This study investigates VGA039, a medicine designed to help clotting by blocking Protein S, an anti-clotting protein. It will assess how VGA039 is absorbed, processed by the body, and how the immune system responds to it.
Objective: To evaluate the effectiveness of VGA039 in preventing bleeding events in people with VWD who have not received routine preventative treatment with VWF-containing concentrates. The study also assesses safety and tolerability.
Endpoints: Primary: Number of bleeding events during the Observational vs. Treatment periods.
Secondary: Bleeding assessments, blood component levels (e.g., haemoglobin), participant feedback, and impact on daily activities.
Study Design: Open-label study with four phases: Screening (up to 9 weeks), Observational (≥24 weeks), Treatment (49 weeks), and optional Extension or Follow-up (8 weeks). Total participation may last up to 142 weeks.
Population: Male and female participants aged 12 to 75 years of age with documented VWD and no other clotting disorders, who have not received routine VWF preventative treatment.
Intervention: VGA039 is administered via subcutaneous injection on a monthly basis following a loading dose. Regular contact with study doctors will occur.
Procedures: Includes medical exams, vital signs, blood/urine tests, ECGs, pregnancy tests, questionnaires, and diaries.
Ethical Considerations: VGA039 has shown good tolerability in prior studies. Possible side effects include injection reactions, headache, flu-like symptoms, thromboembolism, and inflammation. Participants must avoid pregnancy or fathering children during the study.REC name
East Midlands - Derby Research Ethics Committee
REC reference
26/EM/0052
Date of REC Opinion
20 Apr 2026
REC opinion
Further Information Favourable Opinion