What side effects are associated with this type of intervention?

Common treatments for Bullous Pemphigoid include systemic corticosteroids, immunosuppressants, and biologics. Systemic corticosteroids, such as prednisone, can cause side effects like weight gain, hypertension, diabetes, osteoporosis, and increased risk of infections. Immunosuppressants, such as azathioprine and mycophenolate mofetil, may lead to bone marrow suppression, liver toxicity, and increased susceptibility to infections. Biologics, including rituximab, can cause infusion reactions, infections, and potential reactivation of hepatitis B. For treatments similar to Efgartigimod PH20 SC (an FcRn inhibitor), side effects may include mild to moderate reactions at the injection site, headache, and increased risk of infections, although specific data on FcRn inhibitors in BP is limited.

What prior FDA approvals exist?

Bullous Pemphigoid is typically treated with systemic corticosteroids and immunosuppressive agents such as azathioprine and mycophenolate mofetil. More recently, biologics like rituximab, an anti-CD20 monoclonal antibody, have been used off-label for refractory cases. As of now, there are no FDA-approved FcRn inhibitors specifically for Bullous Pemphigoid, but ongoing studies like the one investigating Efgartigimod PH20 SC aim to explore this novel therapeutic approach.

What other types of research exist?

Promising novel alternatives to Efgartigimod PH20 SC for Bullous Pemphigoid patients include: 1) Rituximab, a monoclonal antibody targeting CD20 on B cells, which has shown efficacy in BP. 2) Dupilumab, an IL-4 receptor alpha antagonist, which has been effective in reducing disease activity in BP. 3) Bruton’s Tyrosine Kinase (BTK) inhibitors, which are being investigated for their role in modulating B cell activity and autoantibody production in BP. These alternatives offer different mechanisms of action and have shown potential in clinical settings.

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Immunomodulator

Efgartigimod for Bullous Pemphigoid (BALLAD Trial)

Phase 2 & 3

Waitlist Available

Research Sponsored by argenx

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Participants have clinical signs of BP

Be older than 18 years old

Must not have

Use of BP treatments other than oral corticosteroids (OCS), topical corticosteroids (TCS), conventional immunosuppressants or dapsone

Pregnant or lactating females and those who intend to become pregnant during the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at week 36

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Possible side effects include reactions at the injection site, increased risk of infections due to immune system changes, potential allergic reactions to ingredients in efgartigimod or Prednisone, as well as typical steroid-related side effects like weight gain and mood swings.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I show symptoms of Bell's palsy.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not using BP treatments other than OCS, TCS, immunosuppressants, or dapsone.

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I am not pregnant, breastfeeding, nor planning to become pregnant during the study.

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I cannot take oral corticosteroids due to health reasons.

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I have a different type of pemphigoid or another autoimmune blistering skin condition.

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I currently have an ongoing infection.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at week 36

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at week 36

This trial's timeline: 3 weeks for screening, Varies for treatment, and at week 36 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Proportion of participants who are in complete remission (CR) while receiving efgartigimod PH20 SC or placebo and have been receiving minimal oral corticosteroid (OCS) therapy for ≥8 weeks at week 36

Side effects data

From 2023 Phase 3 trial • 222 Patients • NCT04598451

14%

HYPERTENSION

12%

INSOMNIA

12%

COVID-19

BLOOD LACTATE DEHYDROGENASE INCREASED

HYPERTRIGLYCERIDAEMIA

MYOPATHY

HEADACHE

DEPRESSION

DERMATITIS ACNEIFORM

INJECTION SITE ERYTHEMA

ORAL CANDIDIASIS

NASOPHARYNGITIS

URINARY TRACT INFECTION

LEUKOCYTURIA

INCREASED TENDENCY TO BRUISE

SPINAL COMPRESSION FRACTURE

GLOMERULONEPHRITIS

DRUG ERUPTION

SKIN INFECTION

HYPOPROTEINAEMIA

RESPIRATORY FAILURE

TACHYCARDIA

VISION BLURRED

WEIGHT INCREASED

PEMPHIGUS

ERYSIPELAS

VOMITING

COVID-19 PNEUMONIA

GASTROENTERITIS

PNEUMONIA

DIABETES MELLITUS

100%

80%

60%

40%

20%

Study treatment Arm

Efgartigimod PH20 SC

Placebo PH20 SC

Trial Design

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

efgartigimod PH20 SC

2021

Completed Phase 3

~490

Prednisone

2014

Completed Phase 4

~2500

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Bullous Pemphigoid (BP) is commonly treated with systemic corticosteroids and immunosuppressants, which work by reducing inflammation and suppressing the immune system to prevent the formation of blisters. Efgartigimod PH20 SC, an FcRn inhibitor, is being studied for its ability to reduce pathogenic IgG autoantibodies by blocking the neonatal Fc receptor (FcRn), which is responsible for recycling IgG. This reduction in autoantibodies can help control disease activity and potentially lead to remission. Understanding these mechanisms is crucial for BP patients as it highlights the importance of targeting the underlying immune processes to manage symptoms and improve quality of life.