VIVID-6VGA039 Prophylaxis for Von Willebrand Disease in Adolescents and Adults
VGA039
Blood Coagulation Disorders+7
+ Blood Platelet Disorders
+ Hematologic Diseases
Treatment Study
Summary
Study start date: October 15, 2025
Actual date on which the first participant was enrolled.This clinical study is designed to assess how well a new treatment, VGA039, works in preventing bleeding episodes in adolescents and adults who have Von Willebrand Disease (VWD), a genetic disorder that affects the blood's ability to clot. The study aims to see if VGA039, given as a subcutaneous injection, can reduce the frequency and severity of bleeding. This research is important because VWD can lead to frequent and serious bleeding problems, and finding effective treatments can significantly improve the quality of life for those affected. Participants in the study will first go through an observational period of at least 24 weeks, where their bleeding patterns and treatments are documented. This is followed by about 49 weeks where they will receive regular injections of VGA039. Throughout the study, participants will keep diaries to record details about any bleeding events, such as how often they occur, how long they last, and where they happen. The study will also monitor any side effects experienced during both the observation and treatment periods to ensure the safety of the participants.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.60 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.From 12 to 75 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
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 Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 4 locations
Arkansas Children's Hospital
Little Rock, United StatesOpen Arkansas Children's Hospital in Google MapsScience 37, Inc. (Virtual Clinical MetaSite)
Morrisville, United StatesHemophilia Center of Western PA
Pittsburgh, United StatesWashington Center for Bleeding Disorders
Seattle, United States