Von Willebrand Disease (VWD)
Conditions
Keywords
VWD, von Willebrand Disease, VIVID-6, Vega Therapeutics, Incyte Corporation, VGA039, Latarcibart
Brief summary
This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
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.
Interventions
DRUGVGA039
VGA039 SC administered for 49-weeks during the interventional phase following a 24-week observational period.
Sponsors
Vega Therapeutics, Inc
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
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.
Eligibility
Sex/Gender
ALL
Age
12 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
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 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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of bleeding events [Efficacy] | Time Frame: From 7 days after informed consent until 49 weeks after SC study drug initiation |
Secondary
| Measure | Time frame |
|---|---|
| Incidence of treated bleeding events [Efficacy] | From 7 days from Informed consent until 49 weeks after SC study drug initiation |
| Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including hypersensitivity, anaphylaxis, or anaphylactoid reactions | From informed consent until 49 weeks after SC study drug initiation |
| Plasma concentrations of SC doses of VGA039 | From just prior to the start of study drug administration until 49 weeks after SC study drug initiation |
| Incidence of Anti-drug antibodies to VGA039 | From just prior to the start of study drug administration until 49 weeks after SC study drug initiation |
Countries
Australia, Austria, Brazil, Georgia, Germany, Ireland, Italy, South Africa, United Kingdom, United States
Contacts
CONTACTClinical Trials- Vega Therapeutics
Outcome results
None listed