Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies
This study is a long-term project collecting information from people with a specific type of condition called AQP4+ NMOSD. Researchers want to understand how Alexion C5 inhibitor medicines affect patients' health and daily lives. They will gather details about how patients are doing, their quality of life, and any side effects over several years. The aim is to see the real-world impact of these treatments. If you're over 18, have been diagnosed with AQP4+ NMOSD, and are already taking one of these specific medicines (eculizumab or ravulizumab), you might be able to take part. The study will look back at your health history and then follow your progress for about five years.
At a glance
What is this study about?
Imagine a special health diary where doctors keep track of how people with a certain condition are doing with their medicine. That's essentially what this study is. It's called a 'registry' because it collects ongoing information from lots of people over a long time. The condition we're focusing on is a specific type of Neuromyelitis Optica Spectrum Disorder (NMOSD) known as AQP4+ NMOSD. This condition affects the brain and spinal cord.
The main goal of this registry is to understand how well certain medicines, called Alexion C5 inhibitor therapies, work in real life for people with AQP4+ NMOSD. These medicines are designed to help prevent future attacks or 'relapses' of the condition. Researchers will look at how the medicines affect patients' health, their daily lives, and their overall well-being. This includes collecting information on things like how often patients have attacks, what their quality of life is like, and any side effects they might experience.
By gathering this kind of detailed information from many patients over several years, doctors and researchers hope to get a clearer picture of the long-term benefits and safety of these treatments. This helps them understand the best ways to manage AQP4+ NMOSD and improve care for people living with the condition.
Key takeaways
- This study collects information about people with AQP4+ NMOSD already taking specific Alexion C5 inhibitor medicines.
- It's an 'observational' study, meaning you won't change your current treatment or have extra medical procedures.
- The goal is to understand the long-term benefits and safety of these medicines in real-world settings.
- Information will be collected for approximately five years.
- You must be over 18 and have a confirmed AQP4+ NMOSD diagnosis to join.
Who may be eligible?
To join this study, you need to be at least 18 years old. You must also have a confirmed diagnosis of AQP4+ NMOSD, which is a specific type of Neuromyelitis Optica Spectrum Disorder.
It's important that you are already receiving one of the Alexion C5 inhibitor medicines for a period of time to help prevent your condition from flaring up. Specifically, you must have had at least one dose of eculizumab in the last four weeks or at least one dose of ravulizumab in the last twelve weeks before joining. The doctors will also need to be able to look back at your health records, including your history of taking these medicines and any relapses you've had in the year before starting the medicine.
You won't be able to take part if you are currently in another study where you are receiving a new experimental drug treatment for AQP4+ NMOSD.
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 18 years old or older?
- Do you have a confirmed diagnosis of AQP4+ NMOSD?
- Are you currently receiving eculizumab (within the last 4 weeks) or ravulizumab (within the last 12 weeks)?
- Can your doctor access your medical history, including medicine doses and past relapses from 1 year before starting your current treatment?
- Are you *not* currently taking part in another study where you're receiving an experimental drug for NMOSD?
What does participation involve?
If you decide to take part, your doctor will look back at your medical information from up to one year before you started your Alexion C5 inhibitor medicine up until today. This includes details about your diagnosis, how you've been treated, and any relapses you've experienced. After this initial review, your health information will be collected regularly while you continue your treatment.
This is an 'observational' study, which means you won't be asked to change your current treatment, take new medicines, or do anything different from your usual care. The doctors will simply collect information on how you are already doing with your prescribed medicine. The information gathering for this registry is expected to continue for about five years from when the last person joins the study. There are no extra visits or assessments beyond your usual appointments with your specialist.
Potential risks and benefits
Locations (33)
- Clinical Trial SiteVerified postcodeWashington D.C., United States· Recruiting
- Research SiteVerified postcodeBoston, United States· Recruiting
- Clinical Trial SiteVerified postcodeChapel Hill, United States· Not yet recruiting
- Clinical Trial SiteVerified postcodeColumbus, United States· Recruiting
- Clinical Trial SiteVerified postcodePlano, United States· Recruiting
- Clinical Trial SiteVerified postcodeMilwaukee, United States· Recruiting
- Clinical Trial SiteVerified postcodeBuenos Aires, Argentina· Recruiting
- Clinical Trial SiteVerified postcodeBuenos Aires, Argentina· Not yet recruiting
- Clinical Trial SiteVerified postcodeBuenos Aires, Argentina· Recruiting
- Clinical Trial SiteVerified postcodeBuenos Aires, Argentina· Recruiting
- Clinical Trial SiteVerified postcodeBurnaby, Canada· Recruiting
- Clinical Trial SiteVerified postcodeBeijing, China· Not yet recruiting
Common questions
What is NMOSD?
NMOSD stands for Neuromyelitis Optica Spectrum Disorder, a rare, lifelong autoimmune disease that affects the brain, spinal cord, and optic nerves.
What does 'observational' mean for this study?
It means you continue your usual treatment, and doctors just collect information about your health over time, without asking you to do anything extra or change your medicine.
Will I take a new medicine in this study?
No, this study is for people who are already taking specific Alexion C5 inhibitor medicines. You won't be given any new or experimental drugs.
How long will I be in the study?
The information collection about your health will continue for approximately five years from when the last person joins the study. You will be followed for as long as you are part of the study.
Why is this study important?
By collecting real-world information about how Alexion C5 inhibitor medicines work, this study helps doctors understand the long-term benefits and safety, which can improve care for people with AQP4+ NMOSD.
How to find out more
Alexion Pharmaceuticals, Inc. (Sponsor)
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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