VGA039 for Von Willebrand Disease

Recruiting at 22 trial locations

Overseen By Clinical 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

Compensation: Varies

Number of Visits: Unknown

116 Participants Needed

Trial Summary

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 VGA039 safe for humans?

The safety of recombinant von Willebrand factor (rVWF), also known as vonicog alfa, has been confirmed in clinical studies for various uses, including on-demand treatment, surgery, and regular prevention. It has shown minimal adverse events, such as allergic reactions and a theoretical risk of blood clots, but more studies are needed for certain groups like pregnant women and children. ^{1 2 3 4 5}

What is the purpose of this trial?

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.

Eligibility Criteria

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.

Timeline

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

Treatment Details

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).

Participant Groups

Group I: Part 5 Experimental Treatment 0 Interventions

Multiple doses of VGA039 in open label extension

Group II: Part 4 Experimental Treatment 1 Intervention

Cohorts of VGA039 single dose for surgical prophylaxis

Group III: Part 3 Experimental Treatment 1 Intervention

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

Group IV: Part 2 Experimental Treatment 1 Intervention

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

Group V: Part 1 Placebo Group 2 Interventions

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

Who Is Running the Clinical Trial?

Vega Therapeutics, Inc

Lead Sponsor

Trials

1

Recruited

120+

Findings from Research

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]

References

1. England 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. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

An association of candidate gene haplotypes and bleeding severity in von Willebrand disease (VWD) type 1 pedigrees. [2021]

3. England 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. [2023]

4. Japan pubmed.ncbi.nlm.nih.gov

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

5. Spain pubmed.ncbi.nlm.nih.gov

Recombinant von Willebrand factor: a first-of-its-kind product for von Willebrand disease. [2017]