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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-10-15

Study Completion Date

2028-10-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD

NCT04052698

Von Willebrand Diseases

COMPLETED PHASE3

An Observational Study of Participants With Type 3 Von Willebrand Disease on Prophylactic Standard-of-Care Treatment

NCT06883240

Von Willebrand Disease, Type 3

RECRUITING

Prevent Postpartum Hemorrhage in Women With Von Willebrand Disease: The VWD-WOMAN Trial

NCT04344860

Von Willebrand Diseases Postpartum Hemorrhage

COMPLETED PHASE3

A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients with VWD

NCT06205095

Von Willebrand Diseases

RECRUITING PHASE3

Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease

NCT01602419

Von Willebrand Disease

COMPLETED

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Von Willebrand Disease (VWD)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SEQUENTIAL

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.

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type EXPERIMENTAL

VGA039

Intervention Type DRUG

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

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

VGA039

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

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

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)

Minimum Eligible Age

12 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No