Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD) (NCT07115004) | Clinical Trial Compass
RecruitingPhase 3
Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)
United States60 participantsStarted 2025-10-15
Plain-language summary
This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
Who can participate
Age range12 Years – 75 Years
SexALL
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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)
What they're measuring
1
Incidence of bleeding events [Efficacy]
Timeframe: Time Frame: From 7 days after informed consent until 49 weeks after SC study drug initiation