Recruiting
Phase 3

VGA039

Sponsor:

Vega Therapeutics, Inc

Code:

NCT07115004

Conditions

Von Willebrand Disease (VWD)

Eligibility Criteria

Sex: All

Age: 12 - 70+

Healthy Volunteers: Not accepted

Interventions

VGA039

Study Details

Brief summary:

This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)

Conditions

Von Willebrand Disease (VWD)

Study ID

NCT07115004

Start date

Oct 15, 2025

Status verified date

Apr, 2026

Completion date

Oct, 2028

Anticipated

Primary completion date

Oct, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 70+

Healthy Volunteers: Not accepted

Key Inclusion Criteria

  • 12 to 75 years of age, inclusive
  • No clinically significant laboratory, ECG, or vital signs results
  • Documented diagnosis consistent with VWD of any type
  • Historical annualized bleeding rate (ABR; excluding menstrual bleeds and bleeds under the skin) of both untreated and treated bleeds ≥12 per year
  • Patients with VWD who are judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes
  • Hemoglobin level ≥ 8 g/dL and platelet count ≥ 100 x 109/L at Screening

Key Exclusion Criteria

  • Use of routine prophylaxis of VWF-containing concentrates defined as at least 1 VWF-containing concentrate infusion to prevent or reduce bleeding per week during the previous 6 months prior to screening
  • Planning to initiate routine prophylaxis with VWF-containing concentrates or any other hemostatic treatment during the study
  • Patients with pro-thrombotic disorders or abnormal findings on laboratory thrombophilia evaluation performed at screening or previously documented
  • History of arterial or venous thrombosis, including superficial thrombophlebitis, or embolism
  • Evidence of renal, hepatic, central nervous system, respiratory, cardiovascular disease, cerebrovascular disease, peripheral vascular disease, or metabolic dysfunction
  • Baseline FVIII activity > lower limit of normal (LLN)

Study Design

Enrollment

60 participants

Anticipated

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Multiple Doses of VGA039 after observational run-in

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

Interventions

VGA039

VGA039 SC administered for 49-weeks during the interventional phase following a 24-week observational period.

Primary outcome measure

  • Incidence of bleeding events [Efficacy] [ Time Frame: Time Frame: From 7 days after informed consent until 49 weeks after SC study drug initiation ]

Central Contacts and Locations

Central contacts

Clinical Trials- Vega Therapeutics

650-466-8041medinfo@star-therapeutics.com

Locations

Arkansas Children's Hospital

Recruiting

Little Rock, Arkansas, United States, 72202

Luskin Orthopaedic Institute For Children

Recruiting

Los Angeles, California, United States, 90007

UC Davis Medical Center

Recruiting

Sacramento, California, United States, 95817

University of California San Francisco

Recruiting

San Francisco, California, United States, 94143

Emory University Hospital

Recruiting

Atlanta, Georgia, United States, 30308

Contacts

Emory University Hospital

(404) 686-0277

Children's Healthcare of Atlanta

Recruiting

Atlanta, Georgia, United States, 30329

University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55455

Science 37, Inc. (Virtual Clinical MetaSite)

Recruiting

Morrisville, North Carolina, United States, 27560

Contacts

Science 37 Recruitment Team

1-855-737-3711recruitment@science37.com

Hemophilia Center of Western PA

Recruiting

Pittsburgh, Pennsylvania, United States, 15213

UT Southwestern Children's Hospital

Recruiting

Dallas, Texas, United States, 75235

Washington Center for Bleeding Disorders

Recruiting

Seattle, Washington, United States, 98101

Contacts

Research@WACBD.org

More Information

Sponsor

Vega Therapeutics, Inc

Last update posted

Apr 3, 2026

Last verified

Apr, 2026

Keywords

  • VWD
  • von Willebrand Disease
  • VIVID-6
  • Vega Therapeutics
  • Star Therapeutics
  • VGA039

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Vega Therapeutics, Inc on 2026-04-03.