98 Participants Needed

Efgartigimod for Bullous Pemphigoid

(BALLAD Trial)

Recruiting at 131 trial locations
Ss
Overseen BySabine s Coppieters, MD
Age: 18+
Sex: Any
Trial Phase: Phase 2 & 3
Sponsor: argenx
Must be taking: Oral corticosteroids
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 4 JurisdictionsThis treatment is already approved in other countries

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

The participant is willing and able to understand the requirements of the study
Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
I am of legal age and can consent to participate.
See 4 more

Exclusion Criteria

I am not using BP treatments other than OCS, TCS, immunosuppressants, or dapsone.
I am not pregnant, breastfeeding, nor planning to become pregnant during the study.
You have tested positive for COVID-19.
See 12 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Phase 2 evaluation to provide proof of concept for the therapeutic activity of efgartigimod PH20 SC

26 weeks

Interim Analysis

Interim analysis performed to assess primary and secondary endpoints and confirm sample size for Part B

1-2 weeks

Treatment Part B

Phase 3 evaluation to confirm results from Part A in a larger group of participants

26 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

10 weeks

Treatment Details

Interventions

  • Efgartigimod PH20 SC
  • Placebo
  • Prednisone
Trial OverviewThe trial is testing efgartigimod PH20 SC against a placebo while also using Prednisone for BP treatment. It has two parts: Phase 2 tests if efgartigimod works (proof of concept), and Phase 3 confirms these results in more people. Participants are randomly chosen to receive either the drug or placebo.
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: efgartigimod PH20 SCExperimental Treatment2 Interventions
participants receiving efgartigimod PH20 SC on top of Prednisone
Group II: placebo PH20 SCPlacebo Group2 Interventions
participants receiving placebo PH20 SC on top of Prednisone

Efgartigimod PH20 SC is already approved in European Union, United States, Japan, China for the following indications:

🇪🇺
Approved in European Union as VYVGART for:
  • Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
🇺🇸
Approved in United States as VYVGART Hytrulo for:
  • Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
  • Chronic inflammatory demyelinating polyneuropathy (CIDP)
🇯🇵
Approved in Japan as VYVDURA for:
  • Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
🇨🇳
Approved in China as Efgartigimod alfa injection (subcutaneous injection) for:
  • 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

Trials
76
Recruited
11,500+

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

Findings from Research

Efgartigimod, an FcRn inhibitor, effectively reduced total IgG and autoreactive IgG levels in patients with pemphigus vulgaris and pemphigus foliaceus, showing early signs of improved disease activity and good tolerability during a phase 2 clinical trial.
Interestingly, while total IgG levels returned to baseline after treatment, autoreactive antibody levels remained low in some participants, suggesting a potential long-lasting immunomodulatory effect of efgartigimod beyond just blocking IgG recycling.
FcRn Antagonism Leads to a Decrease of Desmoglein-Specific B Cells: Secondary Analysis of a Phase 2 Study of Efgartigimod in Pemphigus Vulgaris and Pemphigus Foliaceus.Maho-Vaillant, M., Sips, M., Golinski, ML., et al.[2023]
Efgartigimod, a first-in-class neonatal Fc receptor antagonist, received its first approval in the USA in December 2021 for treating generalized myasthenia gravis in adults who are positive for anti-acetylcholine receptor antibodies.
The drug is also being evaluated for other autoimmune diseases and has been approved in Japan for generalized myasthenia gravis patients regardless of antibody status, indicating its potential broad application in autoimmune conditions.
Efgartigimod: First Approval.Heo, YA.[2022]
Efgartigimod alfa is the first neonatal Fc receptor antagonist approved for treating generalized myasthenia gravis (gMG), showing significant and rapid improvements in muscle strength and quality of life in a phase 3 trial with a placebo group.
The treatment was generally well tolerated, with most side effects being mild to moderate, indicating a favorable safety profile for patients with gMG.
Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use.Heo, YA.[2023]

References

FcRn Antagonism Leads to a Decrease of Desmoglein-Specific B Cells: Secondary Analysis of a Phase 2 Study of Efgartigimod in Pemphigus Vulgaris and Pemphigus Foliaceus. [2023]
"Half-half" blisters in bullous pemphigoid successfully treated with adjuvant high-dose intravenous immunoglobulin. [2014]
Effect of FcRn antagonism on protective antibodies and to vaccines in IgG-mediated autoimmune diseases pemphigus and generalised myasthenia gravis. [2022]
Complete FcRn dependence for intravenous Ig therapy in autoimmune skin blistering diseases. [2023]
Efgartigimod: First Approval. [2022]
Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. [2023]
Fc-binding proteins enhance autoantibody-induced BP180 depletion in pemphigoid. [2023]