A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
This study is investigating a new treatment called efgartigimod for adults with bullous pemphigoid, a skin condition causing large, fluid-filled blisters. Researchers want to see if efgartigimod, given as injections, is safe and effective in helping people achieve clear skin and stay off steroid tablets (like prednisone) for at least 8 weeks. Participants will be randomly assigned to receive either the new medicine or a placebo (a dummy treatment) alongside their usual steroid treatment. The study is particularly interested in how many people achieve complete remission and can reduce or stop their steroid medication. It also looks at how long it takes for the condition to improve and any side effects.
At a glance
What is this study about?
This research study is for adults who have a skin condition called bullous pemphigoid. This condition causes large, itchy blisters on the skin. You might know that treatments often involve steroid tablets, like prednisone, which can have side effects when used for a long time or at high doses.
Researchers are testing a new medicine called efgartigimod. They want to find out if it's safe and if it can help people with bullous pemphigoid achieve clear skin (what doctors call 'complete remission') and then stay well without needing steroid tablets for at least 8 weeks. The study will compare efgartigimod with a placebo, which is a treatment that looks like the real medicine but doesn't contain any active drug. This helps researchers understand if any improvements are genuinely due to the new medicine.
The main goal is to see how many people on efgartigimod can get their skin clear and stay off steroid tablets for a good period. They will also look at how quickly people get better, how much steroid medicine they need, how itchy their skin is, and any side effects. This information will help doctors understand if efgartigimod could be a better treatment option for people with bullous pemphigoid in the future.
Key takeaways
- This study is testing a new medicine called efgartigimod for bullous pemphigoid.
- It aims to see if the new medicine can clear skin and reduce the need for steroid tablets.
- Participants will receive either the new medicine or a dummy treatment, plus prednisone.
- Regular visits and checks will be part of the study participation.
- The study is open to adults aged 18 and over with bullous pemphigoid.
- You can stop participating at any time if you wish.
Who may be eligible?
This study is looking for adults who have been diagnosed with bullous pemphigoid. There's no upper age limit, so if you're 18 years or older, you might be able to take part.
Both men and women can join the study. The doctors and nurses running the study will check your medical history and current health carefully to make sure the study is a good fit for you and that it's safe for you to participate. This might include reviewing any other health conditions you have or other medicines you're taking.
- Are you 18 years old or older?
- Have you been diagnosed with bullous pemphigoid?
- Are you able to attend regular clinic appointments?
- Are you willing to potentially receive either the study drug or a placebo?
- Are you taking any other medications that the study team would need to review?
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'll be randomly assigned to one of two groups: one receiving the new medicine (efgartigimod) and the other receiving a placebo (a dummy treatment). You won't know which group you're in, and neither will your study doctor, to keep the results fair. Both groups will also receive prednisone, a common steroid medicine.
Taking part will involve regular visits to the clinic for check-ups, assessments of your skin, and to receive your study medication, which will be given as injections. Doctors will also track the amount of steroid medicine you're taking. You'll have regular follow-up appointments to monitor your progress, how your skin is healing, and any potential side effects. The total duration of your participation in the study will be explained in detail by the study team.
Potential risks and benefits
Locations (14)
- —Czechia
- —Italy
- —Spain
- —Hungary
- —Slovakia
- —Bulgaria
- —Romania
- —Netherlands
- —Croatia
- —Germany
- —Greece
- —Poland
+2 more sites — see the official record for the full list.
Common questions
What is bullous pemphigoid?
It's a skin condition that causes large, fluid-filled blisters to form on the body.
What is efgartigimod?
It's a new medicine being tested to see if it can help treat bullous pemphigoid.
What is a placebo?
It's a dummy treatment that looks like the real medicine but contains no active drug, used for comparison.
Will I still take my regular steroid medicine?
Yes, participants in both groups will also receive prednisone, a common steroid medicine.
Can I leave the study if I change my mind?
Yes, you can withdraw from the study at any time 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.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.