Efgartigimod for Scleroderma

(eSScape Trial)

Recruiting at 60 trial locations

Overseen By Sabine Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: argenx

Disqualifiers: Pulmonary hypertension, Digital vasculopathy, Scleroderma mimics, 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

Duration: 48 weeks

81 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called efgartigimod PH20 SC to assess its safety and effectiveness for people with systemic sclerosis, a condition that causes skin and organ hardening. Participants will receive either the treatment or a placebo (a substance with no active drug), and the trial will last up to 15 months. Individuals diagnosed with systemic sclerosis and specific symptoms like thickened skin might be suitable candidates. The goal is to determine if this new treatment can help manage systemic sclerosis symptoms. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to potential advancements in managing systemic sclerosis.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that efgartigimod PH20 SC is likely to be safe for humans?

Studies have shown that efgartigimod PH20 SC is generally well-tolerated by patients. In trials with this treatment, more than 10% of participants experienced mild side effects, such as headaches or reactions at the injection site. Efgartigimod PH20 SC is currently undergoing testing in a Phase 2 trial. While some safety information is available, further research is needed to fully understand its safety. For any concerns or questions, discussing them with the study team or a doctor is advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for scleroderma?

Unlike current treatments for scleroderma that focus on managing symptoms or suppressing the immune system in general, efgartigimod PH20 SC represents a novel approach by specifically targeting and reducing pathogenic autoantibodies. This treatment is particularly interesting because it combines efgartigimod, an antibody fragment that decreases harmful antibodies, with PH20, an enzyme that enhances subcutaneous absorption. Researchers are excited about this treatment's potential to offer more precise control over the autoimmune response in scleroderma, possibly leading to better outcomes with fewer side effects compared to broader immunosuppressive therapies.

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

Research suggests that efgartigimod PH20 SC, which trial participants may receive, might help reduce symptoms of systemic sclerosis, a condition that causes skin thickening. Previous studies have shown that this treatment can significantly affect the immune system, which is crucial because systemic sclerosis involves the immune system attacking the body's own tissues. Early findings indicate that efgartigimod PH20 SC may help lower certain antibodies responsible for symptoms in autoimmune diseases. Although more research is needed to confirm its effectiveness for systemic sclerosis, these early signs are promising. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

Adults with systemic sclerosis can join this trial if they're over 18, have had symptoms for less than 5 years (or less than 2 years if positive for a specific autoantibody), and meet certain criteria like skin condition suitable for injections, disability scores, and positive ANA test results. They must also fit the classification criteria for diffuse or limited SSc.

Inclusion Criteria

I have moderate to severe disability or my overall health is rated poorly.

I have at least one area of skin suitable for injection.

Has a positive antinuclear antibodies (ANA) test result at the central laboratory with titer of at least 1:160

Exclusion Criteria

My blood test shows positive for anticentromere antibodies.

My skin is thickening due to a condition similar to or diagnosed as localized scleroderma.

I have severe high blood pressure in the arteries of my lungs.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive efgartigimod PH20 SC or placebo for up to 48 weeks

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Efgartigimod PH20 SC

Trial Overview The trial is testing Efgartigimod PH20 SC against a placebo in adults with systemic sclerosis. Participants will be randomly assigned to receive either the drug or placebo in a ratio of 2:1 during up to a year-long treatment period followed by safety monitoring.

How Is the Trial Designed?

Group I: Efgartigimod PH20 SC Experimental Treatment 1 Intervention

Participants receiving efgartigimod PH20 SC

Group II: Placebo PH20 SC Placebo Group 1 Intervention

Participants receiving placebo PH20 SC

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 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]

Efgartigimod, a new treatment that reduces pathogenic IgG autoantibodies, showed promising results in three patients with both stiff-person syndrome (SPS) and myasthenia gravis (MG) over a 12-week treatment period, leading to symptom improvement.

This study suggests that efgartigimod could be a potential therapy for SPS and other autoimmune neurological disorders, as it demonstrated efficacy in alleviating symptoms associated with both conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome. Di Stefano, V., Alonge, P., Rini, N., et al.[2023]

Efgartigimod, a human IgG1 antibody, significantly improved symptoms in patients with generalized myasthenia gravis, with 68% of treated patients showing a meaningful response compared to only 30% in the placebo group, indicating strong efficacy.

The treatment was well tolerated, with similar rates of adverse events between efgartigimod and placebo groups, suggesting it is a safe option for patients, with no deaths reported during the trial.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Howard, JF., Bril, V., Vu, T., et al.[2022]

Efgartigimod was found to be safe and well-tolerated in a phase 2 study involving 24 patients with generalized myasthenia gravis, with no serious adverse events reported.

The treatment led to a significant reduction in pathogenic IgG autoantibodies and resulted in a rapid and long-lasting improvement in disease symptoms for 75% of patients, suggesting its potential as an effective therapy for myasthenia gravis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. Howard, JF., Bril, V., Burns, TM., et al.[2020]

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 is a promising new treatment for primary immune thrombocytopenia (ITP) that works by binding to the neonatal Fc receptor (FcRn), which leads to increased degradation of IgG antibodies that cause platelet destruction.

This therapy offers a novel mechanism of action compared to existing treatments, potentially providing an effective option for patients who may not respond well to traditional therapies like corticosteroids or IVIgG.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod alfa for the treatment of primary immune thrombocytopenia. Broome, C.[2023]

Citations

1. theglobeandmail.com theglobeandmail.com/investing/markets/stocks/ARGX-Q/pressreleases/33669577/argenxs-promising-phase-2-study-on-efgartigimod-ph20-sc-for-systemic-sclerosis/

Argenx's Promising Phase 2 Study on Efgartigimod PH20 ... ' The study aims to assess the efficacy and safety of efgartigimod PH20 SC in adults with systemic sclerosis, a condition with limited treatment ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06655155

NCT06655155 | A Study to Assess the Efficacy and Safety ... The main purpose of this study is to evaluate the effect and safety of efgartigimod PH20 SC compared to placebo in adults with systemic sclerosis.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06655155?term=NCT06655155&rank=1

A Study to Assess the Efficacy and Safety of Efgartigimod ... The main purpose of this study is to evaluate the effect and safety of efgartigimod PH20 SC compared to placebo in adults with systemic ...

4. argenx.com argenx.com/patients/clinical-trials

Clinical Trials Open-label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod PH20 SC in Participants From 2 to Less ...

5. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-efgartigimod-ph20-sc-for-adults-with-systemic-sclerosis/

Study on the Effectiveness and Safety of Efgartigimod ... This treatment is being compared to a placebo to see how effective it is in reducing skin thickening in people with Systemic Sclerosis. The ...

6. clinicaltrial.be clinicaltrial.be/en/details/458375?per_page=20&only_recruiting=0&only_eligible=0&only_active=0

A Study of the Efficacy and Safety of Efgartigimod in Pat... 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).

7. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/myasthenia-gravis/ean-2024/Long%20Term%20Safety%20and%20Efficacy%20of%20Subcutaneous%20Efgartigimod%20PH20%20Interim%20Results%20of%20the%20ADAPT%20SC+%20Trial.pdf

Interim Results of the ADAPT-SC+ Trial dMost frequent AEs occurring in >10% of participants receiving efgartigimod PH20 SC. Figure 1. Mean Change in MG-ADL From Study Baselinea. AChR- ...