Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)
Purpose
This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
Condition
- Von Willebrand Disease (VWD)
Eligibility
- Eligible Ages
- Between 12 Years and 75 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
Exclusion Criteria
- Use of routine prophylaxis of VWF-containing concentrates defined as at least 1 VWF-containing concentrate infusion to prevent or reduce bleeding per week during the previous 6 months prior to screening - Planning to initiate routine prophylaxis with VWF-containing concentrates or any other hemostatic treatment during the study - Patients with pro-thrombotic disorders or abnormal findings on laboratory thrombophilia evaluation performed at screening or previously documented - 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 - Baseline FVIII activity > lower limit of normal (LLN)
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Sequential Assignment
- Intervention Model Description
- The study includes two sequential phases: an initial observational period, during which participants are monitored without receiving study drug, followed by an interventional period where eligible participants are assigned to receive the investigational product VGA039 for approximately 49 weeks. The interventional phase follows a single-group assignment model.
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Multiple Doses of VGA039 after observational run-in |
Participants who meet eligibility criteria and complete a 24-week observational phase will receive VGA039 SC for approximately 49 weeks. |
|
Recruiting Locations
More Details
- Status
- Recruiting
- Sponsor
- Vega Therapeutics, Inc
Detailed 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 patient diaries. Adverse events will also be monitored and recorded throughout both study periods.