Observatory of Patients With Haemophilia B Treated by IdElvion®
This study, called OrPHEe, is looking at how well a medicine called Idelvion® works for people with haemophilia B in France. Haemophilia B is a bleeding disorder where the blood doesn't clot properly due to a missing blood factor called Factor IX (FIX). Doctors already know Idelvion® helps, as shown in previous studies. This new study is observing patients in their normal lives to see how the treatment performs daily. It collects information on whether the medicine continues to prevent bleeds and if it’s safe, helping to understand its real-world benefits for patients.
At a glance
What is this study about?
This study, called OrPHEe, is for people in France who have haemophilia B and are treated with a medicine called Idelvion®. Haemophilia B is a condition where your blood doesn't clot as it should, making you more likely to bleed. This happens because you don't have enough of a special protein in your blood called Factor IX (FIX).
Treatments for haemophilia B involve giving you the missing Factor IX. Idelvion® is a specific type of Factor IX treatment. Previous studies have already shown that Idelvion® is helpful for children and adults with haemophilia B, whether they use it to prevent bleeds regularly (called prophylaxis), for periods when they have a higher risk of bleeding, or to treat bleeds when they happen. It has also been shown to be useful around surgery.
The main goal of this current study is to simply observe patients in their daily lives. By doing this, doctors want to gather more information to confirm that Idelvion® continues to be effective in preventing bleeding episodes and is safe for patients in real-world situations. This type of study is important because it adds to our understanding of how medicines work outside of very controlled clinical trials.
Key takeaways
- This study looks at how Idelvion® works for people with haemophilia B in their normal lives.
- It aims to confirm the medicine's effectiveness and safety, based on real-world experiences.
- You would continue your Idelvion® treatment as usual, with no new medicines involved.
- Participation helps gather important information to understand and improve care for haemophilia B patients.
- This study is currently recruiting patients in France.
Who may be eligible?
To join this study, people must have haemophilia B and be currently using, or have previously used, Idelvion®. This could be for regular treatment to prevent bleeds, to treat bleeds when they happen, or for short periods around surgery or higher risk.
Taking part is voluntary. You (or your parents, if you're under 18) must agree after reading about the study. It's important that you don't have certain types of antibodies (special proteins) against Factor IX in your blood when you join.
There are also reasons why someone couldn't join. You can't take part if you (or your legal representative) don't want to. Also, if your doctor knows that Idelvion® isn't suitable for you (for example, if you have allergic reactions to Factor IX or hamster proteins), you wouldn't be able to participate. Lastly, if you're already in another study where you're trying a new medicine, you can't be in this one too.
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.
- I have been diagnosed with haemophilia B.
- I am currently using or have used Idelvion® for my treatment.
- I (or my parents if I'm under 18) would agree to take part after getting information.
- My doctor has not said I have any allergies to Idelvion® or similar medicines.
- I am not currently taking part in another study testing a new medicine.
What does participation involve?
This study is an 'observational' study. This means you would continue to receive your Idelvion® treatment as prescribed by your doctor, just as you normally would. The study isn't asking you to take a new or different medicine. Doctors will simply record information about your health and how your Idelvion® treatment is working during your usual clinic visits. There are no extra tests or visits specifically for this study. The total duration of your participation would depend on how long you remain on Idelvion® and your doctor continues to collect this information.
Potential risks and benefits
Locations (28)
- CHU Amiens - PicardieVerified postcodeAmiens, France
- CH Annecy GenevoisVerified postcodeAnnecy, France
- CHRU BesançonVerified postcodeBesançon, France
- CHU Bordeaux - Hôpital PellegrinVerified postcodeBordeaux, France
- CHRU BrestVerified postcodeBrest, France
- CHU CaenVerified postcodeCaen, France
- CHU Clermont-FerrandVerified postcodeClermont-Ferrand, France
- CHU DijonVerified postcodeDijon, France
- Hôpital Simone VeilVerified postcodeEaubonne, France
- CHU de GrenobleVerified postcodeGrenoble, France
- CRC-MHC Hôpital Kremlin BicêtreVerified postcodeLe Kremlin-Bicêtre, France
- CH du MansVerified postcodeLe Mans, France
Common questions
What is haemophilia B?
Haemophilia B is a blood disorder where your blood doesn't clot properly because you have too little of a certain protein called Factor IX (FIX).
What is Idelvion®?
Idelvion® is a medication that provides the missing Factor IX protein to help your blood clot.
Will I take a new medicine in this study?
No, you will continue your Idelvion® treatment as prescribed by your doctor. This study just observes how it works in real life.
How long will I be in the study?
Your participation will be tied to how long you are treated with Idelvion® and as long as your doctor is collecting this information as part of the study.
Do I have to live in France to participate?
Yes, this particular study is being done with patients in France.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.