RecruitingOBSERVATIONAL

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

This study is for people with haemophilia B. It's called an 'observational' study, meaning researchers will watch and collect information about how people are doing over time, rather than trying a new treatment in a controlled way. The main goal is to understand how well a new gene therapy called HEMGENIX works and if it's safe for people with haemophilia B, both shortly after treatment and for many years to come. The study will also look at a group of patients who are receiving a standard treatment called Factor IX (FIX) prophylaxis. This comparison will help doctors understand the benefits and any potential downsides of HEMGENIX more clearly. It's about gathering real-world information to help future patients.

At a glance

Status

Recruiting

Sponsor

CSL Behring

Enrolment target

500

Start

15 Jun 2023

Estimated completion

01 Aug 2043

Hemophilia B

What is this study about?

This study wants to learn more about a treatment called HEMGENIX for people who have haemophilia B. Haemophilia B is a rare inherited condition where your blood doesn't clot properly, leading to longer bleeding after an injury or surgery, and sometimes bleeding inside the body.

HEMGENIX is a type of gene therapy. This means it aims to help your body produce the missing clotting factor (Factor IX) itself, rather than needing regular injections of it. Because HEMGENIX is a newer treatment, doctors want to carefully watch people who receive it to understand how well it works over many years and if there are any long-term side effects or safety concerns. This study is like following a group of people over time to see how the treatment affects their health and quality of life.

The study also includes another group of people with haemophilia B who are having standard treatment with Factor IX (FIX) shots. By comparing the HEMGENIX group with the FIX group, researchers can get a clearer picture of how HEMGENIX performs compared to what's already available. This helps ensure that doctors have the best possible information when advising patients about their treatment options.

Key takeaways

This study observes real-world use of HEMGENIX for haemophilia B.
It aims to understand long-term effectiveness and safety.
It also collects data on standard Factor IX prophylaxis for comparison.
Participation involves sharing medical information from routine care.
It's for adults (18+) with haemophilia B.
The findings will help improve future treatment decisions for haemophilia B.

Who may be eligible?

To be part of this study, you would need to be 18 years old or older. There are two main groups of people the study is observing.

One group is for people who have already had the HEMGENIX treatment commercially, meaning they received it as a standard medical treatment, not as part of a clinical trial. You would need to agree to join the study either shortly before or within six months of having your HEMGENIX treatment, or within six months of when your local clinic starts participating in the study.

The other group is for adult patients with haemophilia B who are currently receiving regular injections of Factor IX (FIX) to prevent bleeding. For this group, you would need to already be part of a specific health registry, like ATHN Transcends Haemophilia Cohort, or a similar one.

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.

Do you have haemophilia B?
Are you 18 years of age or older?
Have you recently received HEMGENIX treatment, or are you planning to?
Or, are you currently on regular Factor IX (FIX) prevention treatment and part of a specific health registry?

Answer every question to see your result.

What does participation involve?

This is an observational study, which means you won't be given a new treatment specifically for the study. Instead, researchers will collect information from your regular medical check-ups and records. If you've received HEMGENIX, they'll monitor how you're doing over time. This involves reviewing your health records from your doctor visits, which will include details about any bleeding episodes you have, how much Factor IX you might still need (if any), and any side effects you experience. For those on standard Factor IX treatment, similar information will be gathered. The study aims to follow participants for a long time to understand the long-term effects of treatments.

Potential risks and benefits

Because this is an observational study, you won't be taking a new experimental drug, so the risks are generally associated with sharing your health information. All personal data will be kept confidential. Potential benefits include helping doctors and researchers understand more about haemophilia B treatments, especially HEMGENIX, which could improve care for future patients. You have the right to withdraw from the study at any time without affecting your medical care.

Locations (12)

American Thrombosis and Hemostasis Network
Verified postcode
Rochester, United States· Recruiting
Medical University Vienna
Verified postcode
Vienna, Austria· Recruiting
Aarhus Universitetshospital
City only
Århus N, Denmark· Recruiting
Centre Hospitalier Universitaire de Brest / CHU Morvan
Verified postcode
Brest, France· Recruiting
Centre Régional de Traitement de l'Hémophilie
Verified postcode
Nantes, France· Recruiting
CHU Nancy - Hôpital Brabois
Verified postcode
Vandœuvre-lès-Nancy, France· Recruiting
Klinik für Angiologie/ Hämostaseologie
Verified postcode
Berlin, Germany· Recruiting
University of Clinic Bonn
Verified postcode
Bonn, Germany· Recruiting
Klinikum der Johann-Wolfgang Goethe Universitaet
Verified postcode
Frankfurt, Germany· Recruiting
Hannover Medical School
Verified postcode
Hanover, Germany· Recruiting
Hospital Alvaro Cunqueiro Dr. Manuel Rodriquez-Lopez
Verified postcode
Vigo, Spain· Recruiting
University Hospital Bern Inselspital
Verified postcode
Bern, Switzerland· Recruiting

Common questions

What is haemophilia B?

Haemophilia B is a genetic condition where your blood doesn't clot properly due to a lack of a specific protein called Factor IX. This can cause prolonged bleeding.

What is HEMGENIX?

HEMGENIX is a gene therapy that aims to help people with haemophilia B produce their own Factor IX, potentially reducing the need for regular injections.

Will I have to take new medicine for this study?

No, this is an 'observational' study. You will continue with your current medical care, and the researchers will simply gather information about your health.

How long will I be part of this study?

This study looks at long-term effects, so it will follow participants for several years to understand how treatments work over time.

What's the difference between this study and a clinical trial?

In an observational study, doctors watch what's already happening and record it. In a clinical trial, people are often given a new treatment to test its effects.

How to find out more

Trial Registration Coordinator

clinicaltrials@cslbehring.com +1 610-878-4697 Official trial record

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