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Active not recruitingOBSERVATIONAL

Effectiveness and Tolerability of Eqwilate in Real-life Conditions

This study, called ONE-TO-ONE, is investigating a medication named Eqwilate for individuals diagnosed with Von Willebrand's Disease (VWD). The main goal is to understand how Eqwilate is used in everyday situations, how effective it is at managing VWD, and if it causes any side effects. It’s looking at people aged 6 years and older. Patients can have any type of VWD and might have used Eqwilate before or be new to the treatment. The researchers want to get a complete picture of Eqwilate's performance outside of strict lab conditions, to see how it truly helps people living with this bleeding disorder.

At a glance

Status
Active not recruiting
Sponsor
Octapharma
Enrolment target
38
Start
27 Nov 2019
Estimated completion
01 Nov 2026

What is this study about?

This research study, known as ONE-TO-ONE, is focused on a medication named Eqwilate, which is used to treat Von Willebrand's Disease. Von Willebrand's Disease is a condition where your blood doesn't clot as well as it should, which can lead to easy bruising and bleeding.

The researchers want to see how Eqwilate works for people in their everyday lives. This means they are looking at how often people need to take it, how effective it is in preventing or stopping bleeding, and if it causes any unwanted side effects when used in a normal setting, not just in very controlled hospital tests. Eqwilate combines two important blood clotting factors, VWF and FVIII, which are often missing or not working properly in people with Von Willebrand's Disease.

Understanding how medicines perform in 'real-life' is very important. It helps doctors and patients know what to expect from a treatment outside of clinical trials, providing a clearer picture of its overall benefits and any practical challenges. This study will help improve our knowledge of how Eqwilate supports people living with Von Willebrand's Disease.

Key takeaways

  • The study focuses on Eqwilate for Von Willebrand's Disease.
  • It aims to understand real-life effectiveness and side effects.
  • Participants must be 6 years or older and currently using Eqwilate.
  • Having any type of VWD is fine, regardless of previous treatment.
  • It helps doctors understand how the medicine works in everyday situations.

Who may be eligible?

To be part of this study, you need to be at least 6 years old and currently receiving treatment with Eqwilate for Von Willebrand's Disease. This means you could be using Eqwilate regularly to prevent bleeding, or perhaps just when you need it, for example, before an operation or when you have an unexpected bleed.

It doesn't matter what specific type of Von Willebrand's Disease you have, nor does it matter if you've been taking Eqwilate for a long time or if you've only just started. The study is open to both men and women.

There are no specific reasons listed that would prevent you from joining this study, as long as you meet the age and treatment criteria.

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.

  1. Are you 6 years old or older?
  2. Do you have Von Willebrand's Disease?
  3. Are you currently being treated with Eqwilate?
  4. Are you happy to share information about your Eqwilate treatment with your medical team?
Answer every question to see your result.

What does participation involve?

The information provided does not detail specific visits, assessments, or the overall duration of participation. However, generally, if you take part in a study like this, it would involve providing information about your experience with Eqwilate. This might include details about how you take the medicine, how well it controls your bleeding, and any side effects you might notice.

This information would likely be collected by your doctor or nurse during your regular appointments. You would continue to receive your Eqwilate treatment as prescribed by your doctor. The study aims to gather data from patients under their usual treatment conditions, meaning it probably wouldn't involve extra medication or different doses than what you're already receiving.

It's important to discuss with your doctor exactly what your involvement would look like, including how often you'd be asked for information and for how long.

Potential risks and benefits

Taking part in a study like this could offer potential benefits by helping researchers better understand how Eqwilate works in everyday life, which could ultimately benefit other people with Von Willebrand's Disease. For you, the direct benefits might include closer monitoring from your care team. Potential risks are generally low as you would be continuing your prescribed treatment with Eqwilate. Any side effects you experience are usually related to the medication itself, rather than your participation in the study. Remember, you have the right to withdraw from the study at any time without it affecting your medical care.

Locations (9)

  • Caen Study Site
    Verified postcode
    Caen, France
  • Clamart Study Site
    Verified postcode
    Clamart, France
  • Paris Study Site - Lariboisiere
    Verified postcode
    Paris, France
  • Paris Study Site - Necker
    Verified postcode
    Paris, France
  • Rennes Study Site
    Verified postcode
    Rennes, France
  • Rouen Study Site
    Verified postcode
    Rouen, France
  • Saint Denis Study Site - Pediatrie
    Verified postcode
    Saint-Denis, France
  • Saint Denis Study Site
    Verified postcode
    Saint-Denis, France
  • Saint-Etienne Study Site
    Verified postcode
    Saint-Etienne, France

Common questions

What is Von Willebrand's Disease?

It's a condition where your blood doesn't clot properly, leading to easier bruising and bleeding than usual.

What is Eqwilate?

Eqwilate is a medicine used to treat Von Willebrand's Disease. It contains important clotting factors that your blood needs.

Why is this study called ONE-TO-ONE?

The study title is a catchy name for 'Modalities of use, effectiveNEss and TOlerability of Eqwilate® a balanced combInatiON of VWF and FVIII in von WillEbrand patients in real-life conditions: the ONE-TO-ONE study'.

Is this study testing a new medicine?

No, this study is looking at how an existing medicine, Eqwilate, works for people in their everyday lives.

Does being in the study mean I'll get different treatment?

No, you would continue with your usual Eqwilate treatment as prescribed by your doctor.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Discussion

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