Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)

NCT07115004 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: VGA039-CP002
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 4

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)

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Incidence of bleeding events [Efficacy]

  Time Frame: From 7 days after informed consent until 49 weeks after SC study drug initiation

Secondary Outcomes (4)

• Incidence of treated bleeding events [Efficacy]

  From 7 days from Informed consent until 49 weeks after SC study drug initiation

• Incidence, nature and severity of adverse events (AEs) and serious adverse events (SAEs), including hypersensitivity, anaphylaxis, or anaphylactoid reactions

  From informed consent until 49 weeks after SC study drug initiation

• Plasma concentrations of SC doses of VGA039

  From just prior to the start of study drug administration until 49 weeks after SC study drug initiation

• Incidence of Anti-drug antibodies to VGA039

  From just prior to the start of study drug administration until 49 weeks after SC study drug initiation

Study Arms (1)

Multiple Doses of VGA039 after observational run-in

EXPERIMENTAL

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

Drug: VGA039

Interventions

VGA039 DRUG

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

Multiple Doses of VGA039 after observational run-in

Eligibility Criteria

• Age: 12 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Vega Therapeutics, Inc: lead

Study Sites (4)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

Science 37, Inc. (Virtual Clinical MetaSite)

Morrisville, North Carolina, 27560, United States

RECRUITING

Hemophilia Center of Western PA

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

Washington Center for Bleeding Disorders

Seattle, Washington, 98101, United States

RECRUITING

MeSH Terms

Conditions

von Willebrand Diseases

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Clinical Trials- Vega Therapeutics

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The study includes two sequential phases: an initial observational period, during which participants are monitored without receiving study drug, followed by an interventional period where eligible participants are assigned to receive the investigational product VGA039 for approximately 49 weeks. The interventional phase follows a single-group assignment model.

Study Record Dates

• First Submitted: August 4, 2025
• First Posted: August 11, 2025
• Study Start: October 15, 2025
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): October 1, 2028
• Last Updated: December 4, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)