Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
This important study is designed to understand the long-term safety and effectiveness of a new medicine called fitusiran. It's for men aged 12 and over who have haemophilia A or B, regardless of whether they have developed certain antibodies. Researchers want to observe how well fitusiran prevents all types of bleeding episodes, including spontaneous and joint bleeds, over an extended period. The study will also explore if this medicine helps improve the daily lives and overall well-being of participants aged 17 and above. The goal is to gather comprehensive information to help decide if fitusiran is a safe and beneficial long-term treatment for haemophilia.
At a glance
What is this study about?
This study is a long-term look at a new medicine called fitusiran, designed for people with haemophilia. Haemophilia is a condition where your blood doesn't clot properly, leading to longer bleeding after an injury or surgery, and sometimes unexpected bleeding inside the body, such as in joints. The main aim of this study is to closely watch the medicine over a long time to make sure it's safe and doesn't cause too many side effects. Researchers will also be checking how well it works to prevent bleeding episodes.
Beyond just preventing bleeds, this study is also interested in how fitusiran might affect your daily life. For those aged 17 and over, researchers will ask questions about your quality of life – meaning how you feel day-to-day, your ability to do activities, and your overall well-being. This helps us understand the full impact of the medicine, not just on your physical health but also on how you experience life with haemophilia.
Taking part in this study means you'd be contributing to valuable research that could help many others with haemophilia in the future. It's about finding better ways to manage the condition and improve health outcomes for those affected.
Key takeaways
- This is a long-term study looking at the safety and effectiveness of a medicine called fitusiran.
- It's for men with severe haemophilia A or B who have taken fitusiran before.
- Researchers want to see how well it prevents bleeding and improves daily life.
- Participation could last up to 7.5 years, with regular medical check-ups.
- You have the right to leave the study at any time.
- It's important to discuss this study thoroughly with your doctor.
Who may be eligible?
This study is looking for men who are at least 12 years old. You must have severe haemophilia A or B, and you should have already taken part in another fitusiran study in the past.
There are also some reasons why you might not be able to join. For example, if you've recently had surgery (within the last 14 days) or are currently getting treatments for clotting problems after surgery, you wouldn't be able to participate. Also, if you're already in a study about gene therapy or if you're regularly using other treatments to prevent bleeding (unless it's just during a study break), you wouldn't be able to join.
The research team will also check your liver function and your medical history for things like blood clots. These are important to make sure the study medicine would be safe for you. The study doctor will go through all the detailed requirements with you.
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 a man aged 12 years or older.
- I have severe haemophilia A or B.
- I have previously taken part in a fitusiran study.
- I have not had surgery in the last 14 days and am not currently receiving extra clotting factor for surgery.
- I am not in a gene therapy trial or regularly using other medicines to prevent bleeds (unless it's just during a study break).
What does participation involve?
If you decide to take part in this study, you could be involved for quite a long time – up to nearly 7 and a half years (90 months). This includes an initial check-up period, the time you'll be taking the study medicine, and a follow-up period to monitor your health. During this time, you will have regular visits to the clinic so the research team can monitor your health, check for any side effects, and see how the medicine is working. You will be provided with the study medicine, fitusiran, and instructions on how to use it. The specific schedule of visits and assessments will be explained in detail by the study team.
Potential risks and benefits
Locations (79)
- Phoenix Childrens Hospital Site Number : 8400009Verified postcodePhoenix, United States
- Children's Hospital Los Angeles Site Number : 8400019Verified postcodeLos Angeles, United States
- Center for Inherited Blood Disorders (CIBD) Site Number : 8400016Verified postcodeOrange, United States
- Nemours Children's Clinic Site Number : 8400008Verified postcodeJacksonville, United States
- St Joseph's Children's Hospital of Tampa Site Number : 8400002Verified postcodeTampa, United States
- Rush University Medical Center -1725 W Harrison St Site Number : 8400001Verified postcodeChicago, United States
- ~Massachusetts General Hospital Site Number : 8400011Verified postcodeBoston, United States
- University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012Verified postcodeAnn Arbor, United States
- Alliance for Childhood Diseases Site Number : 8400007Verified postcodeLas Vegas, United States
- Childrens Hospital Medical Center of Akron Site Number : 8400006Verified postcodeAkron, United States
- Investigational Site Number : 0360001Verified postcodeCamperdown, Australia
- Investigational Site Number : 0360003Verified postcodePrahran, Australia
Common questions
What is haemophilia?
Haemophilia is a genetic condition that prevents blood from clotting properly, which can lead to longer bleeding after injuries or spontaneous bleeding.
What is fitusiran?
Fitusiran is a new medicine being studied to see if it can help prevent bleeding episodes in people with haemophilia.
How long will I be in the study?
You could be in the study for up to 90 months, which is about 7 and a half years, including screening, treatment, and follow-up.
Will I have to pay to be in the study?
No, you will not have to pay for the study medicine or study-related visits and tests.
Can women take part in this study?
No, this particular study is only open to men aged 12 and above.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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