A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
This research is a follow-up study for men with severe Haemophilia A who have already received a treatment called BMN 270 in an earlier clinical trial. The main goal is to understand the long-term effects of this treatment. We're looking at how safe it is over many years, well beyond the initial five-year check-up. We also want to see how well it continues to work in managing their Haemophilia A. This information helps us understand if the benefits of BMN 270 last for a very long time, offering valuable insights for future treatments.
At a glance
What is this study about?
Imagine you've been part of a study for a new treatment for Haemophilia A called BMN 270. This new study is designed to keep checking in with you to see how you're doing over a much longer period. Think of it like a long-term check-up to make sure the treatment continues to be safe and effective for many years after you first received it. Researchers want to understand if the positive effects of BMN 270 last well into the future, beyond the first five years of monitoring.
The initial studies looked at the treatment for at least five years. Now, this follow-up study aims to gather information for around ten years in total about people who had BMN 270. This longer timeframe allows medical experts to get a much clearer picture of how the treatment works over time. It helps them understand its long-term safety and whether its benefits continue to help reduce bleeding and improve quality of life for those with severe Haemophilia A.
This kind of long-term research is really important. It helps doctors and researchers understand the full picture of a new treatment's impact, not just in the short term, but for many years to come. This information helps shape future care for people living with Haemophilia A.
Key takeaways
- This study tracks the long-term effects of BMN 270 for severe Haemophilia A.
- It checks how safe and effective the treatment remains for over ten years.
- Only men (aged 18+) who received BMN 270 in a previous study can join.
- You can still be in the study if you're on other Haemophilia A treatments.
- Participation helps future patients by providing vital long-term data.
Who may be eligible?
This study is for adult men (aged 18 and over) who have severe Haemophilia A and have previously taken part in a study where they received BMN 270. You can join this long-term follow-up study even if you've started other treatments for your Haemophilia A since receiving BMN 270.
To be included, you need to be able to understand and sign a consent form, agreeing to follow the study's instructions. If you're unable to provide consent yourself, a legal representative can do it for you. We are looking for people who are generally healthy, without other serious conditions that might make it hard to follow the study or affect the results.
There are a few reasons why someone might not be able to join. For example, if you finished your previous BMN 270 study a long time ago (more than 4 months) and didn't sign up for this follow-up straight away, you might need special permission to join. Also, you can't be part of the study if you are currently in an institution because of a court order.
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 an adult male (18 or older)?
- Do you have severe Haemophilia A?
- Did you receive BMN 270 in a previous clinical trial?
- Are you able to understand and sign consent forms?
- Are you generally healthy, without other serious conditions?
- Were you enrolled in this follow-up study within 4 months of your previous study ending (or have discussed this with your doctor)?
What does participation involve?
If you decide to take part in this follow-up study, you'll be monitored for approximately ten years in total, from the time you first received BMN 270. This will involve regular visits where doctors will check your health and how your Haemophilia A is being managed. They will gather information about any health changes, any bleeding episodes you might have, and any other treatments you might be using for your Haemophilia A. The exact number and frequency of visits will be explained to you, but the main aim is to keep track of your well-being and the long-term effects of the BMN 270 treatment. You won't receive BMN 270 again in this particular study, as it's purely for long-term observation.
Potential risks and benefits
Locations (41)
- Orthopedic Institute for Children, Orthopedic Hemophilia Treatment CenterVerified postcodeLos Angeles, United States
- UC Davis Hemophilia Treatment CenterVerified postcodeSacramento, United States
- University of California San Diego, Hemophilia & Thrombosis Treatment CenterVerified postcodeSan Diego, United States
- University of California San Francisco - Moffit Hospital, Outpatient Hematology ClinicVerified postcodeSan Francisco, United States
- UCN Hemophilia and Thrombosis CenterVerified postcodeShandon, United States
- Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical CampusVerified postcodeAurora, United States
- St Joseph's Children's Hospital, Center for Bleeding and Clotting DisordersVerified postcodeTampa, United States
- University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer CenterVerified postcodeLouisville, United States
- University of Michigan, Pediatric Hematology and OncologyVerified postcodeAnn Arbor, United States
- Children's Hospital of Michigan, Hemophilia and Thrombosis CenterVerified postcodeDetroit, United States
- Washington University School of Medicine, Department of Pediatrics, Division of Hematology/OncologyVerified postcodeSt Louis, United States
- Nationwide Children's HospitalVerified postcodeColumbus, United States
Common questions
What is Haemophilia A?
Haemophilia A is a rare inherited bleeding disorder where your blood doesn't clot properly, leading to longer bleeding after injuries or surgery, and sometimes internal bleeding.
What is BMN 270?
BMN 270 is a treatment that was given in an earlier clinical trial for severe Haemophilia A, and this study aims to see how it works over many years.
Why is this study important?
It's important because it helps us understand if the good effects and safety of BMN 270 last for a very long time, which helps improve future treatments.
Do I have to stop my current Haemophilia A treatment to join?
No, you don't. You can still participate in this follow-up study even if you've started receiving other treatments for your Haemophilia A.
What if I change my mind about participating?
You are completely free to leave the study at any time, for any reason, without it affecting your medical care.
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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