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116 Participants Needed

VGA039 for Von Willebrand Disease

Recruiting at 24 trial locations

Overseen ByClinical Trials

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Vega Therapeutics, Inc

Must not be taking: Hormonal contraceptives

Disqualifiers: Thrombosis, Pro-thrombotic disorders, others

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.¹ ² ³ ⁴ ⁵

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.¹ ² ⁵ ⁶ ⁷

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

Additional important requirements for participants in the second part of the study.

I have Von Willebrand Disease and experience bleeding or bruising.

I have not used hormonal contraceptives in the last 56 days.

See 6 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Randomized, double-blind, placebo-controlled, single-ascending dose evaluation of IV or SC VGA039 or placebo in healthy volunteers

15 weeks (IV) or 8 weeks (SC)

Treatment Part 2

Open-label, single-ascending dose of SC or IV VGA039 in subjects diagnosed with VWD

15 weeks (IV) or 8 weeks (SC)

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

15 weeks (IV) or 8 weeks (SC)

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

Trial Overview

The study tests VGA039's safety and effects when given once via IV or SC to healthy volunteers and VWD patients. It's a Phase 1a trial, meaning it's an early-stage study focusing on how the drug behaves in the body (pharmacokinetics) and its impact on the disease (pharmacodynamics).

How Is the Trial Designed?

Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 5Experimental Treatment0 Interventions

Multiple doses of VGA039 in open label extension

Group II: Part 4Experimental Treatment1 Intervention

Cohorts of VGA039 single dose for surgical prophylaxis

Group III: Part 3Experimental Treatment1 Intervention

Cohorts MD-1 to MD-4, SC VGA039 multiple doses, dose to be determined

Group IV: Part 2Experimental Treatment1 Intervention

Cohorts A-H IV or SC VGA039 dose to be determined

Group V: Part 1Placebo Group2 Interventions

Cohorts 1-8 IV or SC VGA039 or Placebo dose to be determined

Find a Clinic Near You

Who Is Running the Clinical Trial?

Vega Therapeutics, Inc

Lead Sponsor

Trials

Recruited

120+

Published Research Related to This Trial

A plasma-derived von Willebrand factor concentrate was shown to be effective in treating severe von Willebrand disease, with 89% of treated bleeding episodes resulting in excellent or good outcomes.

The treatment was safe, with no thrombotic complications reported and no development of antibodies against VWF or FVIII in patients, indicating a favorable safety profile for this concentrate.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of severe von Willebrand disease with a high-purity von Willebrand factor concentrate (Wilfactin): a prospective study of 50 patients.Borel-Derlon, A., Federici, AB., Roussel-Robert, V., et al.[2023]

In a post hoc analysis of a phase 3 trial involving 18 adults with type 3 von Willebrand disease, recombinant von Willebrand factor (rVWF) prophylaxis significantly reduced annualized bleeding rates by 91.6% in patients previously treated on-demand, indicating its high efficacy.

The safety profile of rVWF was favorable, with only one non-serious adverse event reported and no serious treatment-related adverse events or development of von Willebrand factor inhibitors, suggesting it is a safe option for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prophylaxis with recombinant von Willebrand factor in patients with type 3 von Willebrand disease: Results of a post hoc analysis from a phase 3 trial.Leebeek, FWG., Peyvandi, F., Tiede, A., et al.[2023]

Vonicog alfa, a recombinant von Willebrand factor product approved in Japan, has shown efficacy and safety in treating von Willebrand disease (VWD) across various clinical scenarios, including on-demand treatment and prophylaxis during surgery.

Clinical studies confirm that vonicog alfa effectively controls bleeding and has a favorable safety profile, with minimal adverse events reported in cases such as treating recurrent nosebleeds and providing prophylaxis for pregnant women.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Advances in the therapy for von Willebrand disease].Nakayama, T.[2023]