What side effects are associated with this type of intervention?

Common treatments for Von Willebrand Disease (VWD) include desmopressin (DDAVP) and vWF replacement therapies. Desmopressin can cause side effects such as headaches, facial flushing, nausea, and, in rare cases, hyponatremia due to water retention. vWF replacement therapies, which involve infusions of plasma-derived or recombinant vWF, can lead to allergic reactions, infusion site reactions, and, rarely, thrombotic events. These side effects are important considerations when managing VWD and should be discussed with a healthcare provider.

What prior FDA approvals exist?

The FDA has approved several treatments for Von Willebrand Disease, primarily focusing on enhancing clotting mechanisms. These include desmopressin (DDAVP), which stimulates the release of von Willebrand factor (vWF) stored in the body, and replacement therapies such as plasma-derived or recombinant vWF concentrates (e.g., Humate-P, Alphanate, and Wilate). These treatments aim to increase vWF levels and improve clotting function, similar to the potential therapeutic approach of VGA039.

What other types of research exist?

One promising alternative to VGA039 for VWD patients is the antibody mAb508, which has shown efficacy in reducing von Willebrand factor (VWF) degradation. Another potential option is the antithrombotic peptide WP-30, which selectively inhibits thrombin-induced platelet aggregation and has demonstrated potent antithrombotic effects in vivo. Both therapies target clotting mechanisms and could be considered for VWD treatment in 2024.

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Monoclonal Antibodies

VGA039 for Von Willebrand Disease

Phase 1

Recruiting

Research Sponsored by Vega Therapeutics, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subjects with VWD who are symptomatic, defined as having a history of bleeding or bruising.

Subjects, 18 to 60 years of age, inclusive.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from start of study drug administration until 15 or 8 weeks after iv or sc study drug administration, respectively

Summary

This trial tests VGA039, a new drug, in healthy people and those with Von Willebrand disease. The goal is to see if it is safe and how it behaves in the body when injected. Participants will be monitored for a period of time after receiving the drug.

Who is the study for?

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.

What is being tested?

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).

What are the potential side effects?

Since this is a Phase 1a trial, specific side effects of VGA039 aren't listed yet as it aims to determine safety and tolerability. However, common side effects in such trials may include injection site reactions, headaches, nausea, fatigue, or allergic responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have Von Willebrand Disease and experience bleeding or bruising.

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I am between 18 and 60 years old.

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I have a genetic condition related to blood clotting.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from start of study drug administration until 15 or 8 weeks after iv or sc study drug administration, respectively

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from start of study drug administration until 15 or 8 weeks after iv or sc study drug administration, respectively

This trial's timeline: 3 weeks for screening, Varies for treatment, and from start of study drug administration until 15 or 8 weeks after iv or sc study drug administration, respectively for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of Treatment-Emergent Adverse Events [Safety and tolerability]

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 2Experimental Treatment1 Intervention

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

Group II: Part 1Placebo Group2 Interventions

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

0 / 3Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Von Willebrand Disease (VWD) treatments primarily focus on correcting the deficiency or dysfunction of von Willebrand factor (VWF), a key protein in blood clotting. Desmopressin (DDAVP) is commonly used to stimulate the release of VWF from blood vessel walls, thereby increasing its levels in the bloodstream. For those unresponsive to DDAVP, VWF concentrates are administered to replace the missing or defective VWF directly. These treatments are crucial for stabilizing blood clot formation and preventing excessive bleeding in VWD patients. The trial VGA039 likely explores a therapeutic agent targeting these clotting mechanisms, potentially offering new or enhanced treatment options.