An Open-label Extension Study of ARGX-113-2009 to Evaluate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
Doctors are studying a new medicine called efgartigimod for adults who have a skin condition called Bullous Pemphigoid. This is an open-label extension study, meaning everyone involved will receive the active treatment. They want to understand how safe the medicine is over a longer period, checking for any side effects. They also want to see how well it works to reduce blisters and help people feel better, especially when used with or without other steroid medications. Researchers will be monitoring participants closely to track how their symptoms change, their quality of life, and if they can stay well without relapses. The main goal is to gather more long-term information on this potential treatment.
At a glance
What is this study about?
This study focuses on a skin condition called Bullous Pemphigoid (BP). BP causes large, fluid-filled blisters to appear on the skin, often in older adults. It can be very uncomfortable and affect day-to-day life. The study aims to learn more about a new treatment, called efgartigimod, given as an injection under the skin. Researchers want to find out how safe the medicine is when used long-term and how well it helps control the blisters and other symptoms of BP.
This particular study is a 'Phase III' study, which means it's a key step to see if the new medicine could become widely available. Because it's an 'open-label' study, all participants will receive the active treatment. This is different from some studies where people might get a dummy treatment. The researchers will be carefully checking for any side effects and how effective the medicine is in helping people go into remission (when symptoms improve or disappear) and stay well.
Ultimately, the goal is to provide a better understanding of how efgartigimod can help people with Bullous Pemphigoid. They'll be looking at how many people achieve remission, how long that remission lasts, if their quality of life improves, and if they can reduce or stop other steroid medicines often used for BP. This information is vital for bringing new and potentially better treatments to those who need them.
Key takeaways
- Looks at long-term safety and effectiveness of efgartigimod for Bullous Pemphigoid.
- All participants receive the active study medicine.
- Aims to reduce blisters and improve quality of life.
- Will assess if other steroid medications can be reduced.
- Involves regular medical visits and assessments.
- Only for adults already in a previous study of efgartigimod.
Who may be eligible?
To join this study, you must be an adult, aged 18 years or older, with a diagnosis of Bullous Pemphigoid. Typically, participants in an 'extension' study like this one have already been involved in an earlier study for the same medicine.
This means that if you haven't been part of the previous efgartigimod study, you likely won't be able to join this specific one. The study is designed to follow up on people who have already started treatment to see how they do over a longer time.
- Are you 18 years old or older?
- Have you been diagnosed with Bullous Pemphigoid?
- Have you previously participated in a study of efgartigimod?
- Are you willing to attend regular visits for medicine and check-ups?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you join this study, you will receive the study medicine, efgartigimod, as an injection under your skin. You'll have regular visits with the study team. These visits will involve checks for any side effects, assessments of your skin blisters using special scores, and discussions about how you're feeling and your quality of life. You may also have blood tests to measure specific markers related to Bullous Pemphigoid and the study medicine. The total duration of your participation will be discussed with you by the study team, but these types of 'extension' studies often involve follow-up over many months or even a few years.
Potential risks and benefits
Locations (10)
- —Bulgaria
- —Italy
- —Czechia
- —Spain
- —Hungary
- —Slovakia
- —Netherlands
- —Germany
- —Croatia
- —Greece
Common questions
What is Bullous Pemphigoid?
It's a rare skin condition that causes large, fluid-filled blisters to form on the skin. It mainly affects older adults.
What is efgartigimod?
It's a new medicine being studied as a potential treatment for Bullous Pemphigoid.
What does 'open-label' mean?
It means everyone in the study will receive the active medicine; no one will get a dummy treatment.
Will I have to stop my current medicines?
The study aims to see if participants can reduce or stop other steroid treatments while on efgartigimod, but this will be carefully managed by the study doctors.
How long does the study last?
This is an 'extension' study, so it will involve follow-up over a longer period, often many months or years. The team will tell you the exact time commitment.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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