A Phase I/IIb extension study assessing the long-term safety and efficacy of an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.
This research is a follow-up study for men who previously received a gene therapy treatment called Hemgenix for severe or moderately severe haemophilia B. The main goal is to check its long-term safety over 6 to 10 years. Researchers will also look at how effective the treatment is, by observing things like how much natural clotting factor (factor IX) the body makes, how often bleeding episodes occur, and if participants still need other treatments. They will also assess how this treatment affects their quality of life. This study builds on earlier research to understand the full picture of this new treatment over many years.
At a glance
What is this study about?
This study is a follow-up to an earlier trial testing a special gene therapy called Hemgenix. It's designed for men who have severe or moderately severe haemophilia B, a condition where the blood doesn't clot properly because of a missing or faulty protein called Factor IX.
The main aim of this follow-up study is to see how safe Hemgenix is in the long term, specifically over 6 to 10 years after people first received the treatment. Researchers will be carefully monitoring participants for any side effects and checking how their bodies are reacting to the treatment. They'll also be looking at detailed blood tests and liver scans to ensure everything is healthy.
Besides safety, the study also wants to understand how well Hemgenix continues to work over these years. This includes checking if the body is still producing enough Factor IX on its own, tracking how often bleeding episodes happen, and seeing if people need less of their usual haemophilia medicines. The study will also explore how the treatment affects their daily lives and overall well-being, helping us understand the full impact of Hemgenix.
Key takeaways
- This is a follow-up study for men previously treated with Hemgenix gene therapy.
- The main goals are to check the long-term safety and effectiveness of Hemgenix over 6 to 10 years.
- Researchers will monitor natural clotting factor levels, bleeding events, and quality of life.
- Participation involves regular check-ups, blood tests, and potentially liver scans.
- This study aims to provide crucial long-term information about Hemgenix for haemophilia B.
Who may be eligible?
To be part of this study, you must be a man who is at least 18 years old. You would have already taken part in the earlier CT-AMT-060-01 study, where you received the Hemgenix treatment for severe or moderately severe haemophilia B.
This study is for participants who have already received the treatment being studied. It's not for new patients to try the treatment for the first time.
If you meet these simple criteria and were in the previous study, you might be eligible to continue helping us understand this important treatment.
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 male?
- Are you 18 years old or older?
- Did you previously take part in the CT-AMT-060-01 study?
- Did you receive the Hemgenix (AAV5-hFIX) treatment in that previous study?
- Do you have severe or moderately severe haemophilia B?
What does participation involve?
If you take part in this study, you will be followed carefully for 6 to 10 years after your initial treatment with Hemgenix. During this time, you will have regular check-ups which will likely involve blood tests to monitor your Factor IX levels and general health. You may also have liver scans (like an ultrasound) to check on your liver health. Researchers will ask you questions about your bleeding episodes, any other medicines you take, and how your haemophilia affects your daily life using questionnaires about your quality of life. The exact number of visits and tests will be explained in detail by the study team, but the purpose is to gather long-term information on the treatment's effects. You will not receive new doses of Hemgenix in this follow-up study.
Potential risks and benefits
Locations (2)
- —UnverifiedNetherlands
- —UnverifiedGermany
Common questions
What is haemophilia B?
Haemophilia B is a genetic condition where your blood doesn't clot properly because you don't have enough of a protein called Factor IX.
What is Hemgenix?
Hemgenix is a type of gene therapy designed to help your body produce its own Factor IX, to improve blood clotting in people with haemophilia B.
Why is this study important?
This study is important because it will show us how safe and effective Hemgenix is over many years, helping us understand its long-term benefits for people with haemophilia B.
Will I receive more treatment in this study?
No, this study is a long-term follow-up for people who already received Hemgenix in a previous trial. You won't receive new doses during this particular study.
What kind of tests will I have?
You'll have regular blood tests to check your Factor IX levels and overall health, and possibly occasional scans of your liver, like an ultrasound.
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.