Efgartigimod for Bullous Pemphigoid

(BALLAD Trial)

No longer recruiting at 146 trial locations

Overseen By Sabine s Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: argenx

Must be taking: Oral corticosteroids

Must not be taking: Investigational products

Disqualifiers: Other pemphigoid, Active infection, Malignancy, others

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, efgartigimod (an experimental drug), for individuals with bullous pemphigoid (BP), a skin condition that causes painful blisters. The goal is to determine if efgartigimod can safely control BP and reduce the need for other medications like steroids. Participants will receive either the new treatment or a placebo (a substance with no active drug). This trial suits adults with moderate to severe BP. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in BP treatment.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that efgartigimod PH20 SC was safe in previous studies. Participants generally tolerated the treatment well, with safety results similar to those of VYVGART, which is already approved for other uses.

Most participants in earlier trials did not experience serious side effects, suggesting that efgartigimod PH20 SC is a safe option for those considering joining a clinical trial. Prospective participants should always consult a healthcare provider about any concerns before deciding to participate. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for bullous pemphigoid, which typically involve corticosteroids like Prednisone, efgartigimod PH20 SC offers a new approach by targeting the neonatal Fc receptor (FcRn). This mechanism effectively reduces the levels of pathogenic IgG antibodies, which are responsible for the symptoms in autoimmune conditions like bullous pemphigoid. Researchers are excited because this targeted action could lead to fewer side effects compared to long-term steroid use, providing a safer and potentially more effective treatment option. Additionally, efgartigimod PH20 SC is administered subcutaneously, which might offer greater convenience for patients compared to intravenous therapies.

What evidence suggests that efgartigimod PH20 SC might be an effective treatment for bullous pemphigoid?

Research has shown that efgartigimod PH20 SC, which participants in this trial may receive, can help manage bullous pemphigoid (BP) by reducing the antibodies that harm the skin. In studies of similar conditions, patients experienced symptom relief and even remission with this treatment. This suggests that efgartigimod PH20 SC might lower the need for high doses of steroids, commonly used to treat BP. The goal is to ease symptoms and possibly achieve remission, making it a promising option for those with moderate to severe BP. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

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.

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.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• Efgartigimod PH20 SC
• Placebo
• Prednisone

Trial Overview The 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.

How Is the Trial Designed?

Group I: efgartigimod PH20 SC Experimental Treatment 2 Interventions

participants receiving efgartigimod PH20 SC on top of Prednisone

Group II: placebo PH20 SC Placebo Group 2 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

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

Published Research Related to This Trial

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. Heo, YA.[2023]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05267600

NCT05267600 | A Phase 2/3 Study of Efgartigimod PH20 ... This study intends to demonstrate that efgartigimod is an effective and safe treatment for BP, providing participants with control of disease activity (CDA) ...

2. argenx.com argenx.com/news/2023/argenx-reports-topline-results-address-study-efgartigimod-sc-pemphigus

argenx Reports Topline Results from ADDRESS Study of ... The ADDRESS results show the proportion of PV patients achieving the primary endpoint of complete remission on a minimal dose of steroids (CRmin) was not ...

3. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/dermatology/pemphigus/society-for-investigative-dermatology/2022/SID%202022_BALLAD%20Poster_FINAL.pdf

Phase 2/3 BALLAD (ARGX-113-2009) Trial Design EFGARTIGIMOD IS CLINICALLY EFFECTIVE AND ... Efgartigimod PH20 SC Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Trial in Bullous Pemphigoid.

4. ctv.veeva.com ctv.veeva.com/study/a-phase-2-3-study-of-efgartigimod-ph20-sc-in-adult-participants-with-bullous-pemphigoid-ballad

A Phase 2/3 Study of Efgartigimod PH20 SC in Adult ... This study intends to demonstrate that efgartigimod is an effective and safe treatment for BP, providing participants with control of disease activity (CDA) ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT05681481

NCT05681481 | A Phase 3 Study to Evaluate the Long- ... The purpose of this study is to evaluate the safety of efgartigimod PH20 SC over a longer period of time in adult participants with moderate-to-severe ...

6. centerwatch.com centerwatch.com/clinical-trials/listings/NCT05681481/a-phase-3-study-to-evaluate-the-long-term-safety-tolerability-and-efficacy-of-efgartigimod-ph20-sc-in-adult-participants-with-bullous-pemphigoid

A Phase 3 Study to Evaluate the Long-term Safety, ... The purpose of this study is to evaluate the safety of efgartigimod PH20 SC over a longer period of time in adult participants with ...