Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)
This research study is investigating a new medication, VGA039, for individuals living with Von Willebrand Disease (VWD). VWD is a lifelong bleeding disorder where your blood doesn't clot properly. The study aims to see if giving VGA039 as an injection under the skin can help prevent bleeding episodes. It's a Phase 3 study, which means it's a key step in testing new medicines before they can be made widely available. Researchers want to understand how safe and effective VGA039 is in reducing how often people with all types of VWD experience bleeds. They will compare a period without the new medicine to a period when participants are taking VGA039. Doctors will carefully monitor participants for any side effects and track how well the medicine works to prevent bleeding.
At a glance
What is this study about?
This study is about a medical condition called Von Willebrand Disease, often shortened to VWD. VWD is a common bleeding disorder that affects how your blood clots. If you have VWD, your body either doesn't make enough of a protein called von Willebrand factor, or the protein doesn't work as it should. This can lead to longer or heavier bleeding, for example, after an injury, surgery, or during periods.
Researchers are testing a new medication called VGA039. The main goal of this study is to see if VGA039, given as an injection under the skin, can help prevent bleeding episodes in people with VWD. This type of treatment, where you take medication regularly to stop problems before they start, is often called 'prophylaxis'. The study will also carefully check for any side effects of VGA039 and compare it to how you manage your VWD without this specific new medicine.
This is a Phase 3 study, which is a very important stage in developing new medications. It means that VGA039 has already been tested in earlier studies. Now, the researchers need more information from a larger group of people to confirm its safety and effectiveness before it might be considered for wider use. By taking part, you could be helping to find a new way to manage VWD.
Key takeaways
- This study is testing a new injectable medication (VGA039) for Von Willebrand Disease.
- It aims to see if VGA039 can prevent bleeding episodes in people with VWD.
- The study has two main parts: an observation period and a treatment period.
- It's a Phase 3 study, meaning it's a key step in developing new medicines.
- You would track your bleeding episodes in a diary throughout the study.
- Participation could last over a year, with regular health checks.
Who may be eligible?
To join this study, people generally need to be between 12 and 75 years old and have a confirmed diagnosis of any type of Von Willebrand Disease (VWD). You would also need to have experienced a certain number of bleeding episodes in the past year (at least 12, not including heavy periods or bruises under the skin), and your doctor should feel that you would benefit from regular treatment to prevent bleeding.
There are also some reasons why someone might not be able to join. For example, if you are already regularly taking other medications containing von Willebrand factor to prevent bleeding, or if you plan to start such treatments during the study, you probably wouldn't be eligible. The study also cannot include individuals with a history of blood clots or certain serious health conditions affecting organs like your kidneys, heart, or brain. Also, your blood tests for a protein called Factor VIII shouldn't be too high.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you between 12 and 75 years old?
- Do you have a confirmed diagnosis of Von Willebrand Disease (any type)?
- Have you had at least 12 bleeding episodes in the past year (excluding heavy periods or skin bruises)?
- Does your doctor think you could benefit from regular treatment to prevent bleeding?
- Do you have any history of blood clots or serious health conditions affecting organs like your heart or kidneys?
What does participation involve?
If you decide to take part in this study, it will involve two main periods. First, there's an 'Observational Period' which lasts at least 24 weeks (about 6 months). During this time, you won't be taking the new study medication, but doctors will collect information about any bleeding episodes you experience and any treatments you use. You'll need to keep a diary to record details about bleeds.
After that, you'll move into the 'Active Treatment Period' which lasts about 49 weeks (around 11-12 months). During this time, you will receive the study medication, VGA039, as an injection under your skin. You'll continue to keep your diary to record any bleeds and what treatments you use. Throughout both periods, the study team will regularly monitor your health, perform blood tests, and check for any side effects. The total duration of your involvement could be over a year.
Potential risks and benefits
Locations (14)
- Arkansas Children's HospitalVerified postcodeLittle Rock, United States· Recruiting
- Luskin Orthopaedic Institute For ChildrenVerified postcodeLos Angeles, United States· Recruiting
- UC Davis Medical CenterVerified postcodeSacramento, United States· Recruiting
- University of California San FranciscoVerified postcodeSan Francisco, United States· Recruiting
- Emory University HospitalVerified postcodeAtlanta, United States· Recruiting
- Children's Healthcare of AtlantaVerified postcodeAtlanta, United States· Recruiting
- University of MinnesotaVerified postcodeMinneapolis, United States· Recruiting
- Science 37, Inc. (Virtual Clinical MetaSite)Verified postcodeMorrisville, United States· Recruiting
- Hemophilia Center of Western PAVerified postcodePittsburgh, United States· Recruiting
- UT Southwestern Children's HospitalVerified postcodeDallas, United States· Recruiting
- Washington Center for Bleeding DisordersVerified postcodeSeattle, United States· Recruiting
- K Eristavi National Center of Experimental and Clinical Surgery (ქირურგიის ეროვნული ცენტრი)Verified postcodeTbilisi, Georgia· Recruiting
Common questions
What is Von Willebrand Disease (VWD)?
VWD is a common bleeding disorder where your blood doesn't clot properly, which can lead to longer or heavier bleeding than usual.
What is VGA039?
VGA039 is a new medication being tested in this study to see if it can help prevent bleeding in people with VWD. It's given as an injection under the skin.
What does Phase 3 mean?
Phase 3 is an important stage of research where a new medication is tested in a larger group of people to confirm its safety and how well it works before it can be considered for wider use.
Will I get the study medication right away?
No, there's an initial period of at least 24 weeks where you won't be taking the study medication, but doctors will gather information about your bleeding episodes.
How long will the study last if I join?
The study involves an observation period of at least 24 weeks, followed by about 49 weeks of taking the study medication, so your total participation could be over a year.
How to find out more
Clinical Trials- Vega Therapeutics
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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