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)
Vega Therapeutics, Inc
Summary
This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
Description
This Phase 3 multicenter, open-label, single-sequence cross-over study will investigate the safety and efficacy of subcutaneous administration of VGA039 as prophylaxis for bleeding in patients with every type of VWD. The study consists of an Observational Period of at least 24 weeks followed by an Active Treatment Period of approximately 49 weeks of VGA039 treatment. Bleeding data and details of treatments used will be collected from each patient during both study periods. The number, duration, location, and types of bleeds experienced, as well as treatments for bleeds, will be recorded in pat…
Eligibility
- Age range
- 12–75 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria * 12 to 75 years of age, inclusive * No clinically significant laboratory, ECG, or vital signs results * Documented diagnosis consistent with VWD of any type * Historical annualized bleeding rate (ABR; excluding menstrual bleeds and bleeds under the skin) of both untreated and treated bleeds ≥12 per year * Patients with VWD who are judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes * Hemoglobin level ≥ 8 g/dL and platelet count ≥ 100 x 109/L at Screening Key Exclusion Criteria * Use of routine prop…
Interventions
- DrugVGA039
VGA039 SC administered for 49-weeks during the interventional phase following a 24-week observational period.
Locations (14)
- Arkansas Children's HospitalLittle Rock, Arkansas
- Luskin Orthopaedic Institute For ChildrenLos Angeles, California
- UC Davis Medical CenterSacramento, California
- University of California San FranciscoSan Francisco, California
- Emory University HospitalAtlanta, Georgia
- Children's Healthcare of AtlantaAtlanta, Georgia