Clinical trial summary

• We are conducting a clinical trial (called Velora Pioneer) for a new investigational drug that could be used to treat people with Von Willebrand Disease.
• In this clinical trial we are recruiting male and female participants aged 18 to 65 years with Von Willebrand Disease.
• We will observe how your body processes the new medicine and how long the new investigational drug stays in your body.
• You will be paid for your time if you take part in this clinical trial.

If you are interested in participating in this patient engagement study:

• The intention of the trial drug is to prevent and reduce bleeding events in people with Type 1 Von Willebrand Disease.
• The clinical trial aims to evaluate the safety of the trial drug when given to people with Type 1 Von Willebrand Disease.
• The trial also aims to look at how long the drug will remain in the body (pharmacokinetics), how it affects the body (pharmacodynamics), and whether it works for preventing bleeding (efficacy).

This clinical trial will be divided into two parts:

Part A: In this part of the trial, different dose levels of the trial drug will be tested. In Part A there will be up to 48 participants. Each participant will receive a single dose of the trial drug. This part will involve a screening period, an in-house stay (during which you will receive the trial drug) and a follow-up period.

Part B: In Part B there will be up to 60 participants. Each participant will receive three doses of the trial drug over a 9 week period. This part will involve a screening period, a treatment period (during which you will receive repeated doses of the trial drug) and a follow-up period.

If you are interested in taking part in Part A of this clinical trial:

• You will need to attend a screening appointment so we can check if you are suitable to take part in the trial. At this appointment, we will perform several tests, including but not limited to taking blood, checking your heart and carrying out a physical examination.
• If results from your screening appointment show you are suitable for the trial, you will be asked to stay for up to 4 nights in our clinical research unit.
• During your in-house stay, you will receive a single dose of the trial drug via an injection under the skin (subcutaneous) into your abdomen, thigh or arm. After the injection, you will be required to return for follow-up visits 8, 15, 29, 48, and 57 days after you receive the trial drug.
• This means the trial will take approximately 12 weeks from your screening visit through to your final visit. However, the trial follow-up duration may be extended based on new data.
• During the trial, we will conduct a number of assessments (like those we did at screening) to check how well you tolerate the trial drug, and to help us understand how long the drug remains in your body (pharmacokinetics) and how it affects your body (pharmacodynamics). If you agree, you will also be asked to complete an electronic diary to record your bleeding events.