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NCT07115004PHASE3Recruiting

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

Vega Therapeutics, Inc

Start Date

10/15/2025

Completion Date

10/1/2028

Summary

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

Detailed Description

This Phase 3 multicenter, open-label, single-sequence cross-over study will investigate the safety and efficacy of subcutaneous administration of VGA039 as prophylaxis for bleeding in patients with every type of VWD. The study consists of an Observational Period of at least 24 weeks followed by an Active Treatment Period of approximately 49 weeks of VGA039 treatment. Bleeding data and details of treatments used will be collected from each patient during both study periods. The number, duration, location, and types of bleeds experienced, as well as treatments for bleeds, will be recorded in patient diaries. Adverse events will also be monitored and recorded throughout both study periods.

Eligibility Criteria

Age Range: 12 years to 75 years

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)

Interventions

DRUG

VGA039

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Conditions

Von Willebrand Disease (VWD)

Locations

Arkansas Children's Hospital

Little Rock, Arkansas 72202

United States

Luskin Orthopaedic Institute For Children

Los Angeles, California 90007

United States

UC Davis Medical Center

Sacramento, California 95817

United States

University of California San Francisco

San Francisco, California 94143

United States

Emory University Hospital

Atlanta, Georgia 30308

United States

Children's Healthcare of Atlanta

Atlanta, Georgia 30329

United States

University of Minnesota

Minneapolis, Minnesota 55455

United States

Science 37, Inc. (Virtual Clinical MetaSite)

Morrisville, North Carolina 27560

United States

Hemophilia Center of Western PA

Pittsburgh, Pennsylvania 15213

United States

UT Southwestern Children's Hospital

Dallas, Texas 75235

United States

Washington Center for Bleeding Disorders

Seattle, Washington 98101

United States

K Eristavi National Center of Experimental and Clinical Surgery (ქირურგიის ეროვნული ცენტრი)

Tbilisi, 0159

Georgia

Frankfurt University Hospital (Universitätsmedizin Frankfurt)

Frankfurt, 60596

Germany

Charlotte Maxeke Johannesburg Academic Hospital School of Pathology Clinical Haematologist

Johannesburg, 2194

South Africa