Efgartigimod for Sjogren's Syndrome

(Unity Trial)

Recruiting at 295 trial locations

Overseen By Sabine Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: argenx

Must not be taking: Cyclophosphamide, Anti-CD20, Anti-CD19

Disqualifiers: Secondary Sjögren's, Active fibromyalgia, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

580 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness of efgartigimod, a medication for individuals with moderate-to-severe Primary Sjögren's Syndrome, an autoimmune disease causing dry eyes and mouth. Participants will initially receive either the medication or a placebo (a treatment with no active drug) without knowing which one they receive. Later, all participants will have the opportunity to receive the actual medication during an open-label period. The trial seeks individuals diagnosed with Primary Sjögren's Syndrome, who experience significant symptoms, and have tested positive for specific antibodies. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking 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 you cannot have used cyclophosphamide within 24 weeks before screening or received anti-CD20 or anti-CD19 antibodies within 6 months before screening.

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

A previous study found efgartigimod to be safe for people with Sjogren's disease. Patients generally tolerated the treatment well, experiencing side effects similar to those in other research. This indicates that efgartigimod is generally safe for humans. However, like any treatment, some side effects may occur, but they are not expected to be surprising based on earlier results. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Sjogren's Syndrome?

Unlike standard treatments for Sjogren's Syndrome, which typically focus on relieving symptoms with drugs like pilocarpine and cevimeline, efgartigimod offers a novel approach by targeting the immune system. Efgartigimod is unique because it is designed to reduce the levels of pathogenic antibodies by inhibiting the neonatal Fc receptor (FcRn). This mechanism can potentially address the underlying autoimmune aspect of Sjogren's Syndrome, rather than just alleviating symptoms. Researchers are excited about efgartigimod because it offers a promising new way to treat the root cause of the condition, potentially improving patient outcomes significantly.

What evidence suggests that efgartigimod might be an effective treatment for Sjögren's Syndrome?

Research has shown that efgartigimod may help treat primary Sjögren's Disease. One study found that 45.5% of patients who took efgartigimod felt better, compared to only 11.1% of those who took a placebo (a harmless pill with no active medicine). In this trial, participants will receive either efgartigimod or a placebo during the double-blinded treatment period. The treatment lowers levels of certain antibodies, called IgG, which are linked to the symptoms of the disease. These findings suggest that efgartigimod could improve the quality of life for people with this condition. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for adults over 18 with moderate-to-severe Primary Sjögren's Disease who meet specific criteria, including a clinESSDAI score of at least 6 and positive Anti-Ro/SS-A. Participants must have some natural salivary flow and consent to the study.

Inclusion Criteria

I meet the specific criteria for a type of joint disease, have active symptoms, tested positive for Anti-Ro/SS-A, and have a certain level of salivary flow.

Exclusion Criteria

I have Sjögren's syndrome as a result of another autoimmune condition.

Active fibromyalgia which is not adequately controlled in the judgment of the investigator, or participant is receiving fibromyalgia treatment that has not been stable treatment for at least 12 weeks before screening

I have not received anti-CD20 or anti-CD19 therapy in the last 6 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Double-blinded Treatment

Participants receive either efgartigimod PH20 SC or placebo during the double-blinded treatment period

48 weeks

Open-label Treatment

Participants receive efgartigimod PH20 SC during the open-label treatment period

Up to 57 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Efgartigimod

Trial Overview The trial is testing Efgartigimod PH20 SC against a placebo in patients with Primary Sjögren's Syndrome. It includes a double-blind period where neither doctors nor patients know who gets what, followed by an open-label phase for up to 105 weeks.

How Is the Trial Designed?

Group I: Open-label treatment period Experimental Treatment 1 Intervention

Participants receiving efgartigimod PH20 SC during the open-label treatment period

Group II: Double-blinded treatment period: Efgartigimod PH20 SC Experimental Treatment 1 Intervention

Participants receiving efgartigimod PH20 SC during the double-blinded treatment period

Group III: Double-blinded treatment period: Placebo PH20 SC Placebo Group 1 Intervention

Participants receiving placebo PH20 SC during the double-blinded treatment period

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

The combination of iguratimod (IGU) and methylprednisolone (MP) significantly reduces markers of inflammation and disease activity in primary Sjögren's syndrome (pSS), including erythrocyte sedimentation rate (ESR) and immunoglobulin levels, based on a meta-analysis of clinical studies.

This treatment combination does not increase the risk of adverse events compared to the standard treatment of hydroxychloroquine (HCQ) and MP, suggesting that IGU and MP may be a safe and effective option for managing pSS.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of iguratimod combined with methylprednisolone for primary Sjögren's syndrome: a meta-analysis and trial sequential analysis. Hu, G., Yu, YF., Yin, S., et al.[2023]

Sjögren's syndrome is often underdiagnosed and undertreated, but recent classification criteria have improved clinical management and research efforts.

The disease is characterized by hypofunction of glandular epithelial cells rather than their destruction, which is crucial for understanding its pathogenesis and guiding treatment options like pilocarpine and hydroxychloroquine.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sjögren's syndrome. Venables, PJ.[2005]

Iguratimod significantly reduces symptoms in patients with Primary Sjogren's syndrome, as evidenced by lower scores in ESSPRI and ESSDAI, and improved Schirmer's test results, based on a meta-analysis of 29 RCTs involving 2258 participants.

The treatment does not appear to increase the risk of adverse events, which were mostly mild, such as gastrointestinal discomfort and rash, suggesting that Iguratimod is a safe option for managing this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Systematic Review and Meta-Analysis of 19 Randomized Controlled Trials of Iguratimod Combined With Other Therapies for Sjogren's Syndrome. Zeng, L., He, Q., Yang, K., et al.[2022]

Cenerimod, a selective sphingosine-1-phosphate receptor 1 modulator, effectively reduced immune cell infiltration and inflammation in salivary glands in two mouse models of Sjögren's syndrome, suggesting its potential to improve gland function.

In both models, cenerimod treatment led to decreased autoantibody production and disaggregation of ectopic lymphoid structures, indicating a reduction in the autoimmune response associated with the disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cenerimod, a selective S1P1 receptor modulator, improves organ-specific disease outcomes in animal models of Sjögren's syndrome. Gerossier, E., Nayar, S., Froidevaux, S., et al.[2021]

In an animal model of Sjögren's Syndrome, Fingolimod effectively reduced the number of circulating T and B cells, which are involved in the autoimmune response, suggesting a potential mechanism for managing the disease.

Fingolimod treatment led to increased salivary output and decreased salivary gland infiltrates, indicating its efficacy in alleviating symptoms associated with Sjögren's Syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fingolimod reduces salivary infiltrates and increases salivary secretion in a murine Sjögren's model. Cohen, PL., McCulloch, A.[2023]

Citations

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

argenx Presents New Efgartigimod Data at EULAR 2025 ... 45.5% of patients receiving efgartigimod achieved improved outcomes on the CRESS composite primary endpoint at Week 24 – including systemic ...

2. pharmatimes.com pharmatimes.com/news/argenx-presents-new-efgartigimod-data-at-eular-2025/

Argenx presents new efgartigimod data at EULAR 2025 45.5% of treated patients showed improvement across key measures, compared to 11.1% of placebo-treated patients.

3. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/eular-june-2025/Efficacy%20and%20Safety%20of%20Efgartigimod%20PH20%20Subcutaneous%20by%20Prefilled%20Syringe%20in%20Adults%20With%20Primary%20Sjogrens%20Disease%20A%20Phase%203_%20Randomized_%20Double-Blind_%20Placebo-Controlled_%20Multicenter%20Trial%20With%20.pdf

Efficacy and Safety of Efgartigimod PH20 Subcutaneous by ... Efficacy and Safety of Efgartigimod PH20 Subcutaneous by Prefilled Syringe in Adults With Primary Sjögren's Disease: A Phase 3, Randomized ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06684847

NCT06684847 | A Study of the Efficacy and Safety ... The main purpose of the proposed study is to evaluate the efficacy of efgartigimod PH20 SC in patients with moderate-to-severe Primary Sjögren's Disease ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0003496725022526

POS0844 EFFICACY AND SAFETY OF EFGARTIGIMOD ... We hypothesize that efgartigimod may successfully reduce disease activity and symptoms in patients with pSjD due to reduction in IgG, including disease-specific ...

6. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/554535

A Study of the Efficacy and Safety of Efgartigimod in Patients ... The main purpose of the proposed study is to evaluate the efficacy of efgartigimod PH20 SC in patients with moderate-to-severe Primary Sjögren's Disease (pSjD).