C0371017 - A PHASE 3, NON-INVESTIGATIONAL PRODUCT, MULTI COUNTRY COHORT STUDY TO DESCRIBE THE LONG-TERM SAFETY AND EFFECTIVENESS OF A PRIOR SINGLE-DOSE TREATMENT WITH INVESTIGATIVE GIROCTOCOGENE FITELPARVOVEC OR FIDANACOGENE ELAPARVOVEC IN PARTICIPANTS WITH HEMOPHILIA A OR HEMOPHILIA B, RESPECTIVELY
This study is called C0371017 and it's all about understanding the long-term effects of special gene therapies for haemophilia A and haemophilia B. These therapies involve a single treatment, and scientists want to follow people who have received them for a long time. The main goal is to check how safe these treatments are and how well they continue to prevent bleeding. Researchers will look for things like blood clots, liver issues, and if the treatment causes the body to react against it. They also want to see if people need less of their usual haemophilia medicine after the gene therapy. This study is for adult males and aims to give doctors and patients a clearer picture of these new treatments.
At a glance
What is this study about?
This study, known as C0371017, is designed to keep a close eye on people who have already received a single gene therapy treatment for haemophilia A or haemophilia B. Think of it as a long-term follow-up to understand how these new treatments work over many years. Haemophilia is a condition where your blood doesn't clot properly, leading to bleeding problems. Gene therapy aims to give your body the instructions it needs to make the clotting factors it's missing.
Researchers want to understand two main things: how safe these gene therapies are in the long run, and how well they continue to help people. Safety is very important, so they will be looking out for any unexpected side effects, such as blood clots, liver problems, or if the body develops a reaction to the treatment. They will also check if the treatment continues to help you make enough clotting factor to prevent bleeds.
Why does this matter? New treatments like gene therapy hold great promise for people with haemophilia, potentially offering a way to reduce or even eliminate the need for regular injections. By carefully following patients over time, this study will provide crucial information to doctors and patients alike, helping them understand the full picture of these therapies beyond their initial effects. This information will help make sure these treatments are as safe and effective as possible for everyone.
Key takeaways
- This study follows people who have received gene therapy for haemophilia A or B.
- The main goal is to check the long-term safety and how well the treatment keeps working.
- Researchers will monitor for side effects like blood clots or liver problems.
- It's for adult males who have already had one of the gene therapies.
- Participation involves regular clinic visits for checks and blood tests.
- The study helps us learn more about these new treatments for the future.
Who may be eligible?
This study is looking for participants who have already received one of the special gene therapy treatments for haemophilia A or haemophilia B. Specifically, it's for those with severe to moderately severe haemophilia B.
You must be at least 18 years old to be considered for this study. There is no upper age limit, meaning adults of any age can potentially take part.
Due to how haemophilia A and B typically affect people, only males can participate in this study.
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 (18 years or older).
- I am male.
- I have haemophilia A or severe to moderately severe haemophilia B.
- I have already received a single gene therapy treatment for my haemophilia.
What does participation involve?
If you join this study, you will be followed over a long period. Since you've already received the gene therapy, the study will involve regular visits to a clinic. During these visits, the study team will take blood samples to check your clotting factor levels and to look for any signs of side effects, like liver changes or blood clots. They will also ask you about your health, any bleeds you've had, and what medicines you are taking. You might also complete questionnaires about your quality of life. The exact number and frequency of visits, as well as the total duration of your participation, will be explained by the study team, but it's typically for many years to track long-term effects.
Potential risks and benefits
Locations (6)
- —UnverifiedItaly
- —UnverifiedGermany
- —UnverifiedFrance
- —UnverifiedSpain
- —UnverifiedSweden
- —UnverifiedGreece
Common questions
What is haemophilia A or B?
Haemophilia is a condition where your blood doesn't clot properly. People with haemophilia A are missing clotting factor VIII, and people with haemophilia B are missing clotting factor IX. This can lead to longer bleeding episodes.
What is gene therapy?
Gene therapy is a new type of treatment that aims to correct the genetic problem causing haemophilia. For haemophilia, it usually involves giving your body new instructions to make the missing clotting factor it needs.
Why is this study necessary if I've already had gene therapy?
This study is important because it helps scientists understand the long-term safety and effectiveness of these new gene therapies over many years, which isn't fully known yet. It helps gather vital information for future patients.
Will I receive any new treatment during this study?
No, this study does not involve giving you any new treatments or medicines. It is a follow-up study to observe the effects of the gene therapy you received in the past.
Will I have to pay to be in this study?
No, participation in a clinical study is usually free, and study-related medical care is covered. You won't have to pay to take part.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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