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 an investigational medicine to study. After that, clinical studies test to see if it is well tolerated 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. 

For more information regarding the study, visit the About BEYOND-9 section.

If you join the study, there are certain things you will be expected to do. This includes, but is not limited to, 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. 

BEYOND-9 may last up to approximately 2 years and 8 months. Participation may last longer (up to 4 years and 2 months) depending on time spent in the lead-in period. Additionally, if you enroll and receive the investigational treatment, you will be asked to join a long-term follow-up study lasting 15 years. 

You will help us increase our medical understanding of hemophilia B which may benefit others in the future with this condition. 

The Informed Consent Form talks about all known risks. Before starting a clinical study, you must sign the document. Signing it means you agree to participate in the research study. The study staff will walk you through the study schedule and design. Ask questions so you are comfortable moving forward. 

Clinical studies are 100% voluntary. You can stop at any time.