Efgartigimod for Immunoglobulin-Related Disease

Overseen By Angie Aberia

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Stanford University

Must not be taking: Immunosuppressives, Glucocorticoids, bDMARDs, others

Disqualifiers: Tuberculosis, HIV, Hepatitis, Cancer, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

5 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether efgartigimod, a new potential drug, can help treat IgG4-related disease, which affects organs like the lacrimal (tear) and salivary glands, and the pancreas. The main goal is to determine if this treatment can reduce swelling in these areas. Participants will receive the medication weekly for up to 12 weeks and will have regular checkups to monitor progress. This trial may suit adults diagnosed with IgG4-related disease, especially those who have experienced discomfort or dryness in their glands and did not respond well to steroids in the past. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

Yes, you will need to stop taking certain medications before joining the trial. Specifically, you cannot be on immunosuppressive medications or certain other drugs like glucocorticoids, csDMARDs, or bDMARDs within 28 days to 6 months before starting the trial, depending on the medication.

Is there any evidence suggesting that efgartigimod is likely to be safe for humans?

Research has shown that efgartigimod is generally well-tolerated in clinical trials for various autoimmune diseases involving IgG antibodies. This treatment appears safe across different dosing schedules. However, patients using efgartigimod, particularly those with conditions like myasthenia gravis, may face a potential risk of infections. Some studies have noted a higher chance of serious infections compared to other treatments. Despite this, efgartigimod continues to show promise as a targeted therapy for diseases like myositis and Sjogren's disease. While efgartigimod appears generally safe, awareness of these infection risks remains important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for IgG4-related disease?

Efgartigimod is unique because it works by reducing levels of pathogenic immunoglobulin G (IgG) antibodies in the body, which are often involved in autoimmune diseases. Unlike traditional treatments that may broadly suppress the immune system, efgartigimod specifically targets the neonatal Fc receptor (FcRn), preventing it from recycling IgG and thus lowering its overall levels. This targeted approach can potentially lead to fewer side effects and more effective management of the disease compared to standard immunosuppressants. Researchers are excited about efgartigimod as it offers a novel mechanism of action and is administered subcutaneously, providing a convenient and potentially more tolerable option for patients.

What evidence suggests that efgartigimod might be an effective treatment for IgG4-related disease?

Research has shown that efgartigimod yields promising results for conditions like IgG4-related disease. In earlier studies, patients who took efgartigimod improved in 4 out of 5 measures of disease activity. Additionally, 80% of these patients experienced very few symptoms, compared to only 14.3% in a comparison group. This suggests that efgartigimod might help reduce symptoms by managing the immune system. While these results are hopeful, further research is needed to confirm its effectiveness specifically for IgG4-related disease. ^{6 7 8 9 10}

Who Is on the Research Team?

Matthew C Baker, MD, MS

Principal Investigator

Stanford University

Are You a Good Fit for This Trial?

This trial is for adults with IgG4-related disease, which affects organs like glands and the pancreas. Participants should be able to visit the clinic regularly for up to 12 weeks and complete questionnaires about their health.

Inclusion Criteria

I have been diagnosed with IgG4-related disease and need treatment.

Have a serum IgG4 concentration greater than or equal to 2 times the upper limit of normal at Screening

Meet the 2019 ACR/EULAR Classification Criteria for IgG4-Related Disease

Exclusion Criteria

I have been treated with an FcRn inhibitor before.

I have used biologic DMARDs within the required timeframes.

I have an autoimmune disease that is not IgG4-related.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive efgartigimod 1000 mg subcutaneously once weekly for up to 12 weeks

12 weeks

Weekly visits for treatment and checkups

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 weeks

Visits every one to six weeks

What Are the Treatments Tested in This Trial?

Interventions

• Efgartigimod

Trial Overview The study tests if efgartigimod can reduce swelling in specific glands or the pancreas in patients with IgG4-related disease. It involves weekly treatments of efgartigimod over a period of up to 12 weeks, with regular clinic visits and health assessments.

How Is the Trial Designed?

Group I: efgartigimod Experimental Treatment 1 Intervention

Participants will be treated with efgartigimod 1000 mg subcutaneously once weekly for up to 12 weeks

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials

2,527

Recruited

17,430,000+

Citations

1. argenx.com argenx.com/news/2025/press-release-3097206

argenx Presents New Efgartigimod Data at EULAR 2025 ... Improvements among patients treated with efgartigimod were achieved in 4 out of 5 CRESS measures. In addition, disease activity among patients ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S1878747925002089

Efficacy and safety of efgartigimod versus intravenous ... The proportion of patients reaching minimal symptom expression (MSE) was 80.0 ​% in the efgartigimod group compared to 14.3 ​% in the IVIg group ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12366048/

The efficacy and safety of efgartigimod for refractory ... Our study demonstrates that efgartigimod is highly effective and well-tolerated in patients with refractory MG. These findings suggest that ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT07025330

A Study of Efgartigimod in Patients With IgG4-Related ... The goal of this clinical trial is to learn if efgartigimod can treat IgG4-related disease in adults. The main questions it aims to answer are:

5. rarediseaseadvisor.com rarediseaseadvisor.com/news/new-study-effectiveness-efgartigimod-igg4-rd/

New Study to Assess Effectiveness of Efgartigimod in IgG4- ... A new study will assess efgartigimod's ability to reduce the volume of the lacrimal gland, salivary gland, and/or pancreas in patients with ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40296516/

Safety profile of efgartigimod from global clinical trials ... Across clinical trials in IgG-mediated autoimmune diseases, efgartigimod was well tolerated with similar safety profiles regardless of dosing regimen.

7. neurology.org neurology.org/doi/10.1212/WNL.0000000000211132

Infections-related Safety Profile of Efgartigimod Alpha and ... This analysis identified a potential risk of infections in myasthenia gravis patients treated with efgartigimod alfa or immunoglobulins in clinical practice.

8. tandfonline.com tandfonline.com/doi/full/10.1080/1744666X.2025.2497840

Safety profile of efgartigimod from global clinical trials ... Across clinical trials in IgG-mediated autoimmune diseases, efgartigimod was well tolerated with similar safety profiles regardless of dosing regimen.

9. neurologylive.com neurologylive.com/view/evaluating-risk-clarifying-safety-data-behind-efgartigimod

Clarifying the Safety Data Behind Efgartigimod In addition, patients on efgartigimod were found to be more likely to have serious infectious events (92% vs 70.65%) and to result in ...

10. argenx.com argenx.com/news/2024/argenx-presents-new-efgartigimod-data-at-eular-2025-highlighting

argenx Presents New Efgartigimod Data at EULAR 2025 ... The data presented at EULAR highlight efgartigimod's potential as a precision therapy for patients living with myositis and Sjogren's disease.