VGA039 for Von Willebrand Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called VGA039 for individuals with Von Willebrand Disease (VWD) and other bleeding disorders. The researchers aim to determine the safety and effectiveness of VGA039 when administered either intravenously or as a subcutaneous injection. The trial includes different groups to explore various doses, including single and multiple doses. Individuals with VWD who experience symptoms like frequent bleeding or bruising might be suitable candidates for this study. As a Phase 1 and Phase 2 trial, this research focuses on understanding how VGA039 works in people and measuring its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking treatment development.
Will I have to stop taking my current medications?
The trial requires that you stop using hormonal contraceptives at least 56 days before starting the study drug. Other medications are not specifically mentioned, so it's best to discuss your current medications with the study team.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that VGA039 is generally safe. In studies, healthy volunteers tolerated VGA039 well. Administering it under the skin was straightforward and did not cause major issues. For individuals with von Willebrand Disease (VWD), VGA039 was also safe and well-tolerated. Specifically, seven VWD patients who used it experienced no serious problems. This suggests that VGA039 could be a safe option for people with bleeding disorders.12345
Why are researchers excited about this trial's treatment?
Researchers are excited about VGA039 for Von Willebrand Disease because it offers a potentially new way to manage this bleeding disorder. Unlike standard treatments like desmopressin or factor replacement therapies, VGA039 is being tested in multiple dosing formats, including both single and multiple dose regimens, which could provide more flexible options for patients. Additionally, VGA039 is being explored for its use in surgical prophylaxis, which could enhance its utility during procedures that pose a bleeding risk. These features may lead to a more tailored and effective approach to managing Von Willebrand Disease, addressing gaps that current treatments might not fully cover.
What evidence suggests that this trial's treatments could be effective for von Willebrand Disease?
Research has shown that VGA039 may help treat von Willebrand Disease (VWD) by boosting thrombin, a key factor in blood clotting. Studies indicate it is safe and easy to use, especially since it can be administered as a shot under the skin rather than through an IV. Early tests in healthy individuals demonstrated its effectiveness and potential convenience for those with VWD. In this trial, researchers are testing VGA039 in various treatment arms, including both single and multiple doses, to evaluate its effectiveness and safety. These findings make VGA039 a promising option for better managing VWD.12567
Are You a Good Fit for This Trial?
This trial is for adults aged 18-60 who are generally healthy or have Von Willebrand Disease (VWD) with a history of bleeding or bruising. Healthy participants must have normal lab results and vital signs, while those with VWD need hemoglobin levels ≥ 8 g/dL and platelet count ≥ 150 × 109/L. People using hormonal contraceptives recently or with certain blood disorders can't join.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part 1
Randomized, double-blind, placebo-controlled, single-ascending dose evaluation of IV or SC VGA039 or placebo in healthy volunteers
Treatment Part 2
Open-label, single-ascending dose of SC or IV VGA039 in subjects diagnosed with VWD
Treatment Part 3
Open-label, Phase 1b study of SC multiple doses of VGA039
Treatment Part 4
Open-label, Phase 2 study of SC single, surgical prophylaxis doses of VGA039 administered prior to a minor surgical procedure
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Multiple doses of VGA039 in eligible subjects diagnosed with VWD who have previously participated in a VGA039 interventional trial
What Are the Treatments Tested in This Trial?
Interventions
- Placebo
- VGA039
Find a Clinic Near You
Who Is Running the Clinical Trial?
Vega Therapeutics, Inc
Lead Sponsor