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Clinical Trials/2025-522056-10-01
2025-522056-10-01
Not yet recruiting
Phase 3

A Prospective, Multicenter, Open-label, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of Prophylactic VGA039 in Adolescent and Adult Patients with von Willebrand Disease (VIVID-6)

Vega Therapeutics Inc.1 site in 1 country2 target enrollmentStarted: December 8, 2025Last updated:

Overview

Phase
Phase 3
Status
Not yet recruiting
Sponsor
Vega Therapeutics Inc.
Enrollment
2
Locations
1
Primary Endpoint
Annualized bleeding rate (ABR)

Overview

Brief Summary

To assess the efficacy of subcutaneous (SC) VGA039 prophylaxis on bleeding events in subjects with von Willebrand Disease (VWD) who previously did not receive von Willebrand Factor (VWF)-containing concentrates on a prophylactic basis

Eligibility Criteria

Ages
18 years to 65+ years (65+ Years, 18-64 Years)
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Male or female subjects, 18-75 years of age, inclusive
  • No clinically significant abnormalities in 12-lead ECG determined at screening.
  • No clinically significant abnormalities in vital signs (blood pressure, pulse rate, respiration rate, oral temperature) determined at screening.
  • Documented diagnosis of and laboratory results consistent with VWD of any type.
  • Subjects must have a historical ABR (excluding menstrual bleeds and bleeds under the skin) ≥ 12 over the 6 months prior to screening.
  • Subjects must be judged by the investigator to be suitable candidates for routine prophylaxis to reduce the frequency of bleeding episodes.
  • Subjects must have a hemoglobin level ≥ 8 g/dL and platelet count ≥ 100 × 10^9/L (or ≥ 50 × 10^9/L if diagnosed with Type 2B VWD) at screening.

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.
  • Detection of FV Leiden or Prothrombin G20210A mutation, or laboratory results consistent with protein C or S deficiency, antithrombin deficiency, or antiphospholipid antibody syndrome at screening
  • Subjects with other known pro-thrombotic disorders or abnormal findings in any prior laboratory thrombophilia evaluation
  • Previous or current treatment for or history of coronary artery diseases, atrial fibrillation, mechanical heart valves or other intravascular foreign materials, venous or arterial thrombosis (Common Terminology Criteria for Adverse Events [CTCAE] Grade >1), or ischemic disease (except for catheter associated thrombosis). Subjects with a positive family history of venous or arterial thromboembolism should also be excluded
  • Severe liver disease (alanine aminotransferase [ALT] and aspartate transaminase [AST] levels >5 times of ULN) or severe kidney disease (estimated glomerular filtration rate [eGFR] < 30 mL/min/1.73 m2)
  • FVIII activity > lower limit of normal (LLN) during Screening. The LLN shall be determined using the assay-specific reference ranges.
  • Subjects who are pregnant or breastfeeding

Outcomes

Primary Outcomes

Annualized bleeding rate (ABR)

Annualized bleeding rate (ABR)

Secondary Outcomes

No secondary outcomes reported

Investigators

Sponsor
Vega Therapeutics Inc.
Sponsor Class
Pharmaceutical company
Responsible Party
Principal Investigator
Principal Investigator

Clinical Trials

Scientific

Vega Therapeutics Inc.

Study Sites (1)

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