A Non-interventional, Post-authorisation Safety Study of Patients Treated With Efgartigimod Alfa
This study is checking the safety of a new medicine, efgartigimod, for a condition called myasthenia gravis (gMG). It's not a typical trial where new treatments are tested; instead, it observes people who are already starting efgartigimod as part of their regular treatment. Doctors want to understand how the medicine works in a real-world setting. Some patients who are starting efgartigimod will be part of the study, and others with gMG who are not taking efgartigimod will also be included to compare. The main goal is to carefully watch for any side effects or safety concerns once the medicine is available for use.
At a glance
What is this study about?
This study is about learning more about a medicine called efgartigimod, which is used to treat a condition called generalized Myasthenia Gravis (gMG). gMG is a long-lasting condition that causes muscle weakness. This particular study is a bit different from typical research trials where new medicines are tested to see if they work. Instead, this study is happening after efgartigimod has already been approved for use, and its main purpose is to carefully watch patients as they take the medicine in their everyday lives.
The main aim is to understand the safety of efgartigimod when it's used by real patients in a real-world setting. This kind of study helps doctors and scientists gather more information about any possible side effects or how people react to the medicine over time, outside of strict clinical trial conditions. It helps ensure that new medicines continue to be safe and effective for everyone who needs them.
To do this, the study will involve two groups of people. One group will be patients with gMG who are either just starting efgartigimod or have recently started it. The other group will be patients with gMG who are not taking efgartigimod and don't plan to start it. By comparing these two groups, researchers can get a clearer picture of how efgartigimod affects patients' health and safety.
Key takeaways
- This study is about the safety of efgartigimod for people with gMG.
- It's an observational study, not a test of a new drug or new treatment.
- You would either be taking efgartigimod or not, depending on your current treatment plan.
- The study helps doctors understand the medicine better in real-world use.
- It's open to adults aged 18 and over with gMG.
- Your regular medical care will not be affected if you choose to participate or not.
Who may be eligible?
To be part of this study, you must be an adult aged 18 or older and have a diagnosis of generalized Myasthenia Gravis (gMG).
If you have gMG, you might be eligible if you are just about to start taking the medicine called efgartigimod, or if you've recently started it within your first course of treatment. Another way you could be eligible is if you have gMG, are not currently taking efgartigimod, and don't have plans to start it.
There are no other specific reasons listed that would stop you from joining this study. Importantly, everyone who joins must agree to take part by signing a consent form.
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?
- Have you been diagnosed with generalized Myasthenia Gravis (gMG)?
- Are you either about to start efgartigimod, or have you recently started it (first cycle)?
- OR, do you have gMG but are not taking efgartigimod and don't plan to start it?
What does participation involve?
This study is designed to observe patients during their regular treatment, rather than adding extra appointments. It doesn't involve new medicines being given specifically for the study. Instead, researchers will collect information from your existing medical records and potentially ask you questions during your routine doctor visits. You will continue to receive efgartigimod (if you are in that group) as prescribed by your doctor. The study will follow you over time to see how you are doing, but the total duration isn't specified, as it's an observational study during your ongoing treatment.
Potential risks and benefits
Locations (35)
- UCSF Medical Center - Helen Diller Family Comprehensive Cancer Center - Brain Tumor Center,Verified postcodeSan Francisco, United States· Recruiting
- SFM Clinical Research, LLCVerified postcodeBoca Raton, United States· Recruiting
- University of Florida College of Medicine JacksonvilleVerified postcodeJacksonville Beach, United States· Recruiting
- Medsol Clinical Research Center IncVerified postcodePort Charlotte, United States· Recruiting
- BayCare Health System, Inc. St Anthony's HospitalVerified postcodeSt. Petersburg, United States· Recruiting
- Prairie Education and Research Cooperative and HSHS Medical GroupVerified postcodeO'Fallon, United States· Recruiting
- University of IowaVerified postcodeIowa City, United States· Recruiting
- University of Kansas Medical CenterVerified postcodeKansas City, United States· Recruiting
- Norton Neuroscience InstituteVerified postcodeLouisville, United States· Recruiting
- Massachusetts General HospitalVerified postcodeBoston, United States· Recruiting
- Beth Israel Deaconess Medical CenterVerified postcodeBoston, United States· Recruiting
- Henry Ford Health SystemVerified postcodeDetroit, United States· Recruiting
Common questions
What is efgartigimod?
Efgartigimod is a medicine used to treat generalized Myasthenia Gravis (gMG), a condition that causes muscle weakness.
Is this study testing a new drug?
No, this study is looking at the safety of efgartigimod after it has already been approved and is being used by patients in their normal lives. It's not testing whether it works.
Will I have to take a new medicine if I join?
No, you will only take efgartigimod if your doctor has already prescribed it for your gMG. The study doesn't ask you to take any new medicines.
What's the difference between the two groups?
One group will be patients who are using efgartigimod, and the other group will be patients with gMG who are not using efgartigimod. This helps researchers compare and understand the medicine's effects.
How long will I be in the study?
The study aims to follow patients over time as part of their regular care. The length of time isn't strictly fixed, as it's about observing ongoing treatment.
How to find out more
Sabine Coppieters, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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