FAQS

A clinical research study is a medical research study in people. The potential new treatment or device being studied only enters a clinical study after researchers test it in the laboratory. If it looks promising, scientists carefully test and collect data about the potential treatment through clinical studies. A clinical study helps us learn how an investigational treatment works in the body. Clinical studies must be reviewed by a regulatory agency. In the United States, it’s the Food and Drug Administration (FDA). In Europe, it’s the European Medicines Agency (EMA). Agencies vary by region. They decide whether to approve the investigational treatment for doctors to prescribe.

The first step is discovering and developing a new investigational medicine to study. After that, clinical studies show if it is safe and effective and provide information on any side effects. Sometimes, researchers compare the potential medicine to a placebo. The placebo looks and is administered like the potential treatment but has no active ingredients. The potential treatment may also be compared to a standard treatment that is already approved and used for treatment. They compare it to current treatments, if available, to find out if the potential treatment is better.

If you join the study, there are certain things you will be expected to do. This includes attending study visits, getting your body weight, height, temperature, blood pressure and respiration rate taken by your study team, getting a scan of your liver to see if it’s healthy, telling your doctor when you experience bleeding that requires additional treatment and getting your blood drawn for some tests. If you are unable to go to your doctor for your study visits, they may be performed at your home.

Your participation may last a minimum of 6 months and up to 2 years with up to 9 study visits.

You may benefit by receiving additional health check-ups on top of your regular medical care. You will also help us increase our medical understanding of haemophilia B which may benefit others in the future with this condition.

Even though you will not receive any study medication in this study, the ICF will give you more information on any possible risks and side effects you may experience when getting your blood drawn or getting a scan of your liver. Your health and safety are our top priorities, and you will be closely monitored during your participation.

You are free to withdraw from the study for any reason and at any time.