Active not recruitingOBSERVATIONAL

A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy

This study is for adult men with Haemophilia B who are already on regular treatment with a medicine called Factor IX. It's not testing a new medicine; instead, it's about understanding how the condition affects people in their daily lives. Researchers want to collect information for at least six months on things like bleeding episodes, how well their current Factor IX treatment works, and how Haemophilia B impacts their quality of life and joint health. This information will help medical experts learn more about the condition, which could lead to better treatments in the future. There's no pressure to join upcoming studies after this one.

At a glance

Status

Active not recruiting

Sponsor

Regeneron Pharmaceuticals

Enrolment target

Start

17 Jan 2024

Estimated completion

21 May 2026

Hemophilia B

What is this study about?

This study is called a 'non-interventional' study, which means you won't be given any new medicines to try. Instead, it's about carefully observing and collecting information from men who already have Haemophilia B and are regularly using their usual Factor IX (FIX) replacement therapy. This therapy is a standard treatment that helps prevent and control bleeding episodes in people with Haemophilia B.

The main goal is to gather detailed information over at least six months about your experiences. This includes understanding how often you have bleeding events, how you use your current FIX treatment, and how different tests measure the FIX in your blood. The study also wants to see if there are any issues with your current treatment, how Haemophilia B affects your overall health and daily life, including your joints, and how often you need medical help for bleeding problems.

This study is an important part of a bigger effort to learn more about Haemophilia B. The information collected will help doctors and researchers better understand the condition. This knowledge could eventually lead to new and better ways to treat Haemophilia B in the future. You will be asked if you'd like to hear about a potential future study involving a new treatment, but you are not obliged to take part.

Key takeaways

This study is for adult men with Haemophilia B already on Factor IX treatment.
It's about understanding your condition and current treatment, not testing new drugs.
You'll continue your usual medical care throughout the study.
Information will be collected for at least six months about your bleeding and health.
Your participation helps improve understanding of Haemophilia B for future treatments.
You can discuss this study with your doctor and decide if it's right for you.

Who may be eligible?

This study is for adult men aged 16 and over who have Haemophilia B.

To join, you must have been using Factor IX treatment for a while (at least 50 times) and be on a steady, regular treatment plan for more than two months. You should also be planning to continue this treatment throughout the study.

However, you can't take part if you've had certain other health problems like a different bleeding disorder, serious liver or heart conditions, or if you've had a strong reaction to Factor IX treatment before. You also can't join if previous tests showed your body makes antibodies against Factor IX, which would make the treatment less effective.

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 an adult male aged 16 or older with Haemophilia B?
Have you been on a regular Factor IX treatment plan for more than 2 months?
Do you plan to continue your Factor IX treatment during the study?
Have you experienced at least 50 doses of Factor IX treatment in the past?
Do you have any serious existing health conditions like advanced liver, heart, or kidney disease?
Have you ever had a serious allergic reaction to Factor IX or developed antibodies against it?

Answer every question to see your result.

What does participation involve?

If you join this study, you will continue your usual Factor IX treatment exactly as prescribed by your doctor. The study will involve collecting information about your health, treatment, and daily experiences for at least six months. This will include keeping track of any bleeding events you have and your regular use of Factor IX. You might be asked to provide blood samples for tests to measure Factor IX activity and check for antibodies. Your quality of life and joint health will also be assessed. The study will also record visits you make to hospitals or doctors for bleeding issues. All of this will be done during your regular medical check-ups and appointments where possible, and you'll typically be followed for between 6 to 12 months.

Potential risks and benefits

A potential benefit of joining this study is contributing valuable information that could help improve future treatments for Haemophilia B, benefiting others with the condition. As you continue your usual care, there are no additional known risks from new medicines, but all medical treatments carry some risk, which your doctor can discuss. You also have the right to withdraw from the study at any time without affecting your medical care.

Locations (17)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of Colorado Hemophilia and Thrombosis Center
Verified postcode
Aurora, United States
Yale HTC
Verified postcode
New Haven, United States
Georgetown University Medical Center
Verified postcode
Washington D.C., United States
University of Florida
Verified postcode
Gainesville, United States
Rush University Medical Center
Verified postcode
Chicago, United States
Indiana Hemophilia and Thrombosis Center
Verified postcode
Indianapolis, United States
University of Michigan
Verified postcode
Ann Arbor, United States
Nationwide Children's Hospital
Verified postcode
Columbus, United States
University of Texas Health Science Center at Houston
Verified postcode
Houston, United States
McMaster University Medical Centre - Hamilton Health Sciences
Verified postcode
Hamilton, Canada
Mcgill University Health Center (MUHC)
Verified postcode
Montreal, Canada
University Hospital of Regensburg
Verified postcode
Regensburg, Germany

Common questions

Will I get a new treatment in this study?

No, this study is about observing and collecting information on your current Factor IX treatment, not trying a new one.

How long will I be involved in the study?

You'll be part of the study for at least six months, and potentially up to 12 months, during which information will be collected.

Will my regular medical care change if I join?

No, you will continue to receive your usual medical care and Factor IX treatment as prescribed by your doctor.

What kind of information will be collected?

The study will collect details on your bleeding episodes, how you use your Factor IX treatment, your quality of life, joint health, and blood test results.

Am I expected to join a future study if I participate in this one?

No, you are not obligated to join any future studies. You will simply be informed about potential future research.