Efgartigimod for Bullous Pemphigoid
(BALLAD Trial)
Trial Summary
What is the purpose of this trial?
This trial tests a new injection treatment called efgartigimod for adults with a severe skin condition called Bullous Pemphigoid. The treatment aims to control the disease by calming down the immune system. The study will check if the treatment is safe and effective in stages, starting with fewer participants and then including more.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it mentions that you should not have an unstable dose of treatments known to affect bullous pemphigoid for at least 4 weeks before starting the trial. You can continue using oral or topical corticosteroids, conventional immunosuppressants, or dapsone.
What data supports the effectiveness of the drug Efgartigimod for treating bullous pemphigoid?
Efgartigimod has shown effectiveness in reducing disease activity in autoimmune conditions like pemphigus, which is similar to bullous pemphigoid, by lowering harmful antibodies. It has also been approved for treating myasthenia gravis, another autoimmune disease, indicating its potential in managing similar conditions.12345
How is the drug Efgartigimod PH20 SC unique for treating bullous pemphigoid?
Efgartigimod PH20 SC is unique because it works by blocking the neonatal Fc receptor (FcRn), which helps reduce harmful antibodies in the body. This mechanism is different from traditional treatments that often rely on steroids, and it may offer a new option for managing bullous pemphigoid with potentially fewer side effects.13567
Eligibility Criteria
Adults with moderate to severe Bullous Pemphigoid (BP) can join this trial. They must understand and follow the study's procedures, agree to use contraception according to local guidelines, and not be pregnant. People with other autoimmune diseases, unstable BP treatments in the last 4 weeks, or a history of certain cancers cannot participate.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part A
Phase 2 evaluation to provide proof of concept for the therapeutic activity of efgartigimod PH20 SC
Interim Analysis
Interim analysis performed to assess primary and secondary endpoints and confirm sample size for Part B
Treatment Part B
Phase 3 evaluation to confirm results from Part A in a larger group of participants
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Efgartigimod PH20 SC
- Placebo
- Prednisone
Efgartigimod PH20 SC is already approved in European Union, United States, Japan, China for the following indications:
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
- Chronic inflammatory demyelinating polyneuropathy (CIDP)
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
Find a Clinic Near You
Who Is Running the Clinical Trial?
argenx
Lead Sponsor
Tim Van Hauwermeiren
argenx
Chief Executive Officer since 2008
B.Sc. and M.Sc. in Bioengineering from Ghent University, Executive MBA from The Vlerick School of Management
Dr. Peter Ulrichts
argenx
Chief Medical Officer since 2023
MD from Maastricht University, PhD in Molecular Immunology from Maastricht University