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A Study of VGA039 to Prevent Bleeding in Teenagers and Adults with von Willebrand Disease Who Have Not Used Regular Preventive Treatment Before

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study is looking at a bleeding disorder called von Willebrand Disease. People with this condition have problems with blood clotting, which means they may experience bleeding episodes more often than usual. The treatment being tested is called VGA039, which is given as an injection under the skin. This medicine is designed to help prevent bleeding from happening in the first place, rather than treating bleeds after they occur.

The purpose of this study is to see how well VGA039 works at reducing the number of bleeding events in people with von Willebrand Disease who have not been using preventive treatment regularly before. The study will also check how safe this medicine is when used over time. During the study, participants will receive regular injections of VGA039 under the skin for a period that can last up to 57 weeks. The doctors will keep track of how many bleeding episodes occur and monitor the overall health of participants throughout the treatment period.

This is a phase 3 study, which means the medicine has already been tested in earlier studies and is now being evaluated in a larger group of people to confirm that it works well and is safe. The study is open-label, which means both the participants and the doctors will know that everyone is receiving the active medicine. Participants will be adolescents and adults with von Willebrand Disease who have experienced frequent bleeding episodes in the past.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial screening and qualification

    Your medical history will be reviewed to confirm a documented diagnosis of von Willebrand Disease, which is a bleeding disorder caused by a problem with a protein in your blood that helps it clot properly.

    A 12-lead ECG (a test that records the electrical activity of your heart) will be performed to ensure there are no significant abnormalities.

    Your vital signs will be measured, including blood pressure, pulse rate, breathing rate, and body temperature.

    Blood tests will be conducted to check your hemoglobin level (a protein in red blood cells that carries oxygen) and platelet count (cells that help your blood clot). Your hemoglobin must be at least 8 grams per deciliter, and your platelet count must be at least 100 × 10^9 per liter (or at least 50 × 10^9 per liter if you have been diagnosed with Type 2B von Willebrand Disease).

    Your bleeding history over the past 6 months will be reviewed. You must have experienced at least 12 bleeding episodes during this period, not counting menstrual bleeding or bruising under the skin.

    The physician will determine whether you are a suitable candidate for regular preventive treatment to reduce how often bleeding episodes occur.

  2. Step 2

    Treatment phase with VGA039

    You will receive VGA039, which is a solution for injection given under the skin (subcutaneous injection).

    The treatment is designed to prevent bleeding episodes by providing regular doses of the medication.

    This phase will continue for an extended period as part of the study's evaluation of how well the medication works and how safe it is.

    The study will track the number of bleeding episodes you experience while receiving the medication.

  3. Step 3

    Ongoing monitoring and assessment

    Throughout the study, your bleeding episodes will be recorded and counted to calculate your annualized bleeding rate, which is the main measure of how well the treatment is working.

    Regular check-ups will be conducted to monitor your health and any potential side effects from the medication.

    Blood tests and other assessments may be performed periodically to ensure the treatment remains safe and effective for you.

Who can join the trial?

9 criteria

  • You must be between 18 and 75 years old
  • You can be male or female
  • You must have a confirmed diagnosis of von Willebrand Disease, which is a bleeding disorder where your blood does not clot properly, and your laboratory test results must support this diagnosis. This can be any type of von Willebrand Disease
  • You must have had 12 or more bleeding episodes in the 6 months before joining the study, not counting menstrual bleeding or bruises under the skin. This is called your annualized bleeding rate
  • Your doctor must believe that you would benefit from regular preventive treatment to reduce how often you have bleeding episodes
  • Your hemoglobin level must be at least 8 g/dL. Hemoglobin is a protein in your red blood cells that carries oxygen throughout your body
  • Your platelet count must be at least 100 × 10^9/L. Platelets are small blood cells that help your blood clot. If you have Type 2B von Willebrand Disease, your platelet count must be at least 50 × 10^9/L
  • Your electrocardiogram, which is a test that measures the electrical activity of your heart, must not show any significant problems
  • Your vital signs, including blood pressure, pulse rate, breathing rate, and body temperature, must not show any significant problems

Who cannot join the trial?

1 criterion

  • No exclusion criteria have been specified in the available study information for this clinical trial.
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Investigated drugs

VGA039 is an investigational medication being studied for the prevention of bleeding in patients with von Willebrand Disease. It is given as an injection under the skin to help reduce the number of bleeding episodes that occur in people with this bleeding disorder.

What is already known about the treatment

VGA039 – VGA039 is an investigational medication currently being studied in phase 3 clinical trials for the prevention of bleeding episodes in patients with von Willebrand Disease. It is administered through subcutaneous injection, meaning it is injected under the skin, and is designed to work as a prophylactic treatment to reduce the frequency of bleeding events. The medication is being evaluated for patients who have not previously received regular preventive treatment with von Willebrand Factor-containing products, and works by helping to improve blood clotting function in individuals whose bodies do not produce enough of this essential clotting protein.

Investigated diseases

von Willebrand Disease – von Willebrand Disease is a bleeding disorder caused by a deficiency or dysfunction of von Willebrand Factor, a protein that helps blood clot properly. People with this condition experience problems with blood clotting, which leads to excessive or prolonged bleeding. Common symptoms include frequent nosebleeds, easy bruising, prolonged bleeding from cuts, and heavy menstrual periods in women. The disease can also cause bleeding in the gums and gastrointestinal tract. The severity of symptoms varies widely among affected individuals, ranging from mild to more significant bleeding episodes. This is an inherited condition that is passed down through families and is considered a rare disease.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2025-522056-10-01Protocol codeVGA039-CP002Estimated enrolment54 patientsSponsorVega Therapeutics Inc.

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