BridgeMD
Phase 3 study of VGA039 prophylaxis in adolescent and adult patients with von Willebrand Disease
EU CTIS ID: 2025-522056-10-01
What this study is testing
To assess the efficacy of subcutaneous (SC) VGA039 prophylaxis on bleeding events in subjects with von Willebrand Disease (VWD) who previously did not receive von Willebrand Factor (VWF)-containing concentrates on a prophylactic basis
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male or female subjects, 18-75 years of age, inclusive
- No clinically significant abnormalities in 12-lead ECG determined at screening.
- No clinically significant abnormalities in vital signs (blood pressure, pulse rate, respiration rate, oral temperature) determined at screening.
- Documented diagnosis of and laboratory results consistent with VWD of any type.
- Subjects must have a historical ABR (excluding menstrual bleeds and bleeds under the skin) ≥ 12 over the 6 months prior to screening.
- Subjects must be judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes.
You likely can't join if
- 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.
- Detection of FV Leiden or Prothrombin G20210A mutation, or laboratory results consistent with protein C or S deficiency, antithrombin deficiency, or antiphospholipid antibody syndrome at screening
- Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation
- Previous or current treatment for or history of coronary artery diseases, atrial fibrillation, mechanical heart valves or other intravascular foreign materials, venous or arterial thrombosis (Common Terminology Criteria for Adverse Events [CTCAE] Grade >1), or ischemic disease (except for catheter associated thrombosis). Subjects with a positive family history of venous or arterial thromboembolism should also be excluded
- Severe liver disease (alanine aminotransferase [ALT] and aspartate transaminase [AST] levels >5 times of ULN) or severe kidney disease (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2)
See the full eligibility criteria
- Male or female subjects, 18-75 years of age, inclusive
- No clinically significant abnormalities in 12-lead ECG determined at screening.
- No clinically significant abnormalities in vital signs (blood pressure, pulse rate, respiration rate, oral temperature) determined at screening.
- Documented diagnosis of and laboratory results consistent with VWD of any type.
- Subjects must have a historical ABR (excluding menstrual bleeds and bleeds under the skin) ≥ 12 over the 6 months prior to screening.
- Subjects must be judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes.
- Subjects must have a hemoglobin level ≥ 8 g/dL and platelet count ≥ 100 × 10^9/L (or ≥ 50 × 10^9/L if diagnosed with Type 2B VWD) at screening.
- 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.
- Detection of FV Leiden or Prothrombin G20210A mutation, or laboratory results consistent with protein C or S deficiency, antithrombin deficiency, or antiphospholipid antibody syndrome at screening
- Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation
- Previous or current treatment for or history of coronary artery diseases, atrial fibrillation, mechanical heart valves or other intravascular foreign materials, venous or arterial thrombosis (Common Terminology Criteria for Adverse Events [CTCAE] Grade >1), or ischemic disease (except for catheter associated thrombosis). Subjects with a positive family history of venous or arterial thromboembolism should also be excluded
- Severe liver disease (alanine aminotransferase [ALT] and aspartate transaminase [AST] levels >5 times of ULN) or severe kidney disease (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2)
- FVIII activity > lower limit of normal (LLN) during Screening. The LLN shall be determined using the assay-specific reference ranges.
- Subjects who are pregnant or breastfeeding
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.