VGA039 for Von Willebrand Disease

(VIVID-6 Trial)

Recruiting at 3 trial locations

Overseen By Clinical Trials- Vega Therapeutics

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Vega Therapeutics, Inc

Must not be taking: VWF concentrates

Disqualifiers: Thrombosis, Renal, Hepatic, Cardiovascular, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness of a new treatment called VGA039, an experimental therapy for individuals with von Willebrand Disease (VWD), a condition that affects blood clotting and causes frequent bleeding episodes. Participants will undergo a 24-week observation period before receiving VGA039 injections for about 49 weeks. The trial seeks individuals with a documented diagnosis of VWD who experience frequent bleeding episodes (12 or more per year, excluding menstrual and under-the-skin bleeds). As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the chance to contribute to potentially groundbreaking treatment advancements.

Do I need to stop my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that VGA039 is likely to be safe for humans?

Research has shown that VGA039 is generally safe. In earlier studies, healthy volunteers tolerated VGA039 well, experiencing no major side effects. Reports also indicate that an injection of VGA039 under the skin reduced bleeding episodes in patients with von Willebrand Disease (VWD).

Although past studies haven't reported any serious side effects, staying informed and discussing any concerns with a doctor is always important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care for Von Willebrand Disease, which often includes replacement therapies or desmopressin, VGA039 is a novel treatment administered subcutaneously. Researchers are excited about VGA039 because it offers a more convenient delivery method compared to traditional intravenous infusions. Additionally, VGA039 may provide a new mechanism of action, potentially targeting the disease more effectively and improving patient outcomes over a longer period. This makes VGA039 a promising advance in the treatment of Von Willebrand Disease.

What evidence suggests that VGA039 might be an effective treatment for Von Willebrand Disease?

Research shows that VGA039 might help people with von Willebrand Disease (VWD) by reducing bleeding. In earlier studies, patients who received VGA039 experienced 75% to 88% fewer bleeding episodes annually, meaning they bled less often over a year. In this trial, participants who meet eligibility criteria and complete a 24-week observational phase will receive VGA039 as a simple shot under the skin for approximately 49 weeks. Early results suggest that VGA039 could be a promising way to manage VWD symptoms. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for people aged 12-75 with Von Willebrand Disease who have a history of frequent bleeding episodes (excluding menstrual and skin bleeds) and are not already on weekly preventive treatments. Participants must have stable vital signs, normal hemoglobin levels, and platelet counts. Those with clotting disorders, past thrombosis, or significant organ disease cannot join.

Inclusion Criteria

Historical annualized bleeding rate (ABR; excluding menstrual bleeds and bleeds under the skin) of both untreated and treated bleeds ≥12 per year

I have been diagnosed with von Willebrand disease.

No clinically significant laboratory, ECG, or vital signs results

Exclusion Criteria

I have been receiving weekly treatments to prevent bleeding for the last 6 months.

Patients with pro-thrombotic disorders or abnormal findings on laboratory thrombophilia evaluation performed at screening or previously documented

I have had blood clots or embolisms in the past.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observational Period

Participants are observed for bleeding events and treatments are recorded

24 weeks

Active Treatment

Participants receive VGA039 SC for prophylaxis of bleeding

49 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VGA039

Trial Overview

The study is testing VGA039 administered under the skin to see if it can reduce bleeding in patients with Von Willebrand Disease. It's a phase 3 trial which means they're looking at how effective this treatment is and monitoring any side effects closely.

How Is the Trial Designed?

Group I: Multiple Doses of VGA039 after observational run-in Experimental Treatment 1 Intervention

Participants who meet eligibility criteria and complete a 24-week observational phase will receive VGA039 SC for approximately 49 weeks.

Who Is Running the Clinical Trial?

Vega Therapeutics, Inc

Lead Sponsor

Trials

1

Recruited

120+

Citations

1.

star-therapeutics.com

star-therapeutics.com/star-therapeutics-presents-clinical-data-for-vga039-a-subcutaneous-therapy-for-the-treatment-of-von-willebrand-disease-vwd/

Star Therapeutics Presents Clinical Data for VGA039, a ...

VGA039 clinical data supports favorable safety profile in healthy volunteers and convenient subcutaneous dosing regimen.

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07115004

Study to Evaluate Subcutaneous (SC) VGA039 in Patients ...

This Phase 3 multicenter, open-label, single-sequence cross-over study will investigate the safety and efficacy of subcutaneous administration of VGA039 as ...

3.

bleeding.org

bleeding.org/news/star-therapeutics-presents-data-on-investigational-vwd-therapy

Interim Trial Data on Investigational VWD Therapy ...

The data showed that a subcutaneous dose of VGA039 was associated with substantial (75% to 88%) reductions in annualized bleed rates in the ...

4.

star-therapeutics.com

star-therapeutics.com/star-therapeutics-to-present-interim-data-from-phase-1-2-multidose-study-of-vga039-in-von-willebrand-disease-at-ash-annual-meeting/

Star Therapeutics to Present Interim Data from Phase 1/2 ...

VGA039 has advanced into a Phase 3 study (NCT07115004), a global single arm cross-over study designed to investigate the safety and efficacy of ...

5.

labiotech.eu

labiotech.eu/trends-news/von-willebrand-disease-treatment-advancements/

Breaking new ground in von Willebrand disease treatment

Explore advances in von Willebrand disease treatment, including Star Therapeutics' VGA039, and other key players in the space.

6.

ashpublications.org

ashpublications.org/blood/article/144/Supplement%201/3981/530216/A-Phase-Ia-Study-of-VGA039-a-Protein-S-Targeting

A Phase Ia Study of VGA039, a Protein S-Targeting ...

Preclinical studies of VGA039 have demonstrated its ability to increase thrombin generation across multiple inherited bleeding disorders, ...

7.

star-therapeutics.com

star-therapeutics.com/star-therapeutics-presents-interim-clinical-data-for-vga039-in-patients-with-von-willebrand-disease-vwd-at-ash-2024/

Star Therapeutics Presents Interim Clinical Data for ...

In single-ascending dose study in VWD patients, a subcutaneous dose of VGA039 was associated with substantial reductions in bleeds.