Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)
This important study is designed for adult men in the UK living with haemophilia B. It's a long-term follow-up for those who previously took part in earlier studies (CSL222_2001 or CSL222_3001) where they received a gene therapy treatment called etranacogene dezaparvovec. The main purpose is to see how safe the treatment remains over many years and how effective it continues to be in managing their haemophilia B. By carefully monitoring participants, researchers hope to gain a better understanding of the lasting effects of this innovative treatment, which could ultimately help improve future care for people with this condition.
At a glance
What is this study about?
This study is a follow-up for adult men with haemophilia B who have already received a special gene therapy. Haemophilia B is a lifelong bleeding disorder where your blood doesn't clot properly due to a missing or faulty ‘Factor IX’ protein. Gene therapy like etranacogene dezaparvovec aims to provide instructions to your body to make this missing protein, potentially reducing bleeding episodes.
This particular study isn't offering new treatment; instead, it's looking at the long-term effects for people who have already had this gene therapy. Researchers want to understand if the treatment continues to be safe over many years and if it keeps working effectively to help manage haemophilia B. This is really important because gene therapies are relatively new, and understanding their long-term impact helps doctors and scientists make informed decisions about their wider use.
By carefully checking on the health and progress of these participants, the study contributes valuable information. It helps build a bigger picture of how this treatment works in the real world, beyond the initial trials. This knowledge is crucial for advancing our understanding of haemophilia B and developing even better therapies in the future.
Key takeaways
- This is a long-term follow-up study for men with haemophilia B.
- Participants have already received a gene therapy called etranacogene dezaparvovec.
- The study aims to understand the long-term safety and effectiveness of the treatment.
- No new medication is given; it involves regular monitoring and health checks.
- Your participation helps improve future care for haemophilia B patients.
Who may be eligible?
To be considered for this study, you must be an adult male who previously participated in one of the original treatment trials for etranacogene dezaparvovec – specifically, study CSL222_2001 or study CSL222_3001.
Additionally, you must have either fully completed your participation in one of those earlier studies, or at least five years must have passed since you received the etranacogene dezaparvovec gene therapy in one of those trials.
There are no other specific reasons listed that would prevent you from joining this follow-up study, provided you meet the criteria above.
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 am an adult male (aged 18 or over).
- I was previously treated with etranacogene dezaparvovec (CSL222) in study CSL222_2001 or CSL222_3001.
- I completed my participation in one of those earlier studies.
- OR at least 5 years have passed since I received the CSL222 treatment.
What does participation involve?
If you decide to take part, you'll be regularly monitored by a medical team. This will involve scheduled visits to the clinic where you'll have various health checks. These typically include blood tests to measure your Factor IX levels and check your general health, physical examinations, and questions about any bleeding episodes or your overall well-being. You won't be receiving new medication as part of this study; it's purely for observation. The total duration of your participation will depend on how long ago you received the gene therapy, as the study aims to track participants for many years after their initial treatment.
Potential risks and benefits
Locations (28)
- 10-15 Phoenix Childrens HospitalVerified postcodePhoenix, United States
- 10-14 Arkansas Children's Hospital - PharmacologyVerified postcodeLittle Rock, United States
- 10-22 Orthopaedic Institute for ChildrenVerified postcodeLos Angeles, United States
- 10-21 Children's Hospital of Los AngelesVerified postcodeLos Angeles, United States
- 10-63 UC Davis Medical CenterVerified postcodeSacramento, United States
- 10-12 University of California, San Diego (UCSD)Verified postcodeSan Diego, United States
- 10-25 University of Colorado DenverVerified postcodeAurora, United States
- 10-19 University of South FloridaVerified postcodeTampa, United States
- 10-10 University of Michigan Medical CenterVerified postcodeAnn Arbor, United States
- 10-16 University of North Carolina at Chapel HillVerified postcodeChapel Hill, United States
- 10-18 Oregon Health & Science UniversityVerified postcodePortland, United States
- 10-20 Vanderbilt University Medical CenterVerified postcodeNashville, United States
Common questions
What is haemophilia B?
Haemophilia B is an inherited bleeding disorder where your blood doesn't clot properly due to a missing or faulty Factor IX protein, leading to prolonged bleeding.
What is etranacogene dezaparvovec?
It's a gene therapy treatment that aims to help your body produce the missing Factor IX protein, which can reduce bleeding episodes in people with haemophilia B.
Am I receiving new treatment in this study?
No, this study is a follow-up. You won't receive new treatment; it's for monitoring those who previously had the gene therapy.
How long will I need to be in the study?
The study aims for long-term follow-up; the duration for you will depend on when you received your initial gene therapy treatment in the parent studies.
What is the purpose of this long-term study?
The main purpose is to understand the long-term safety and effectiveness of etranacogene dezaparvovec gene therapy for men with haemophilia B.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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