Efgartigimod for Myositis

(ALKIVIA Trial)

Not currently recruiting at 463 trial locations

Overseen By Sabine s Coppieters, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: argenx

Disqualifiers: Active infection, Autoimmune disease, Malignancy, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medicine called efgartigimod (specifically, Efgartigimod PH20 SC) to determine its effectiveness for certain muscle diseases, known as idiopathic inflammatory myopathy (IIM). It focuses on conditions such as dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), and some types of polymyositis (PM). Participants will receive either efgartigimod or a placebo to compare effects. Those diagnosed with active muscle disease, such as muscle weakness or specific skin rashes, might be suitable candidates. As a Phase 2, Phase 3 trial, this research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

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 participants must be on a permitted background treatment for idiopathic inflammatory myopathy. It's best to discuss your specific medications with the study team.

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

Research has shown that efgartigimod PH20 SC is generally safe for people with myositis. Earlier studies found it to be as safe and well-tolerated as a placebo. Participants experienced significant improvements, with few serious side effects. The safety data from these studies suggest that efgartigimod PH20 SC could be a promising option with manageable risks for those considering joining a clinical trial. ^{1 2 3 4 5}

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

Unlike standard treatments for myositis, which often involve corticosteroids and immunosuppressants, efgartigimod offers a novel approach by targeting the neonatal Fc receptor (FcRn). This unique mechanism reduces the levels of pathogenic antibodies that contribute to the condition. Additionally, efgartigimod is administered subcutaneously, which can be more convenient for patients compared to intravenous therapies. Researchers are excited because this treatment could potentially offer a more targeted and convenient option with fewer side effects.

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

Research has shown that efgartigimod PH20 SC, which participants in this trial may receive, may help treat idiopathic inflammatory myopathy (IIM). In one study, patients who received efgartigimod showed much better improvement scores compared to those who received a placebo. This suggests the treatment might effectively reduce symptoms of IIM, including conditions like dermatomyositis and polymyositis. The treatment was also well-tolerated, causing few side effects. These findings offer hope for those seeking better management of their IIM symptoms. ^{2 3 4 5 6}

Are You a Good Fit for This Trial?

Adults with active Idiopathic Inflammatory Myopathy (IIM), specifically dermatomyositis, immune-mediated necrotizing myopathy, or polymyositis including antisynthetase syndrome. Participants must have muscle weakness and abnormal enzyme levels indicating IIM. They should be on stable IIM treatments and use contraception if applicable. Exclusions include other primary causes of muscle weakness, severe infections like HIV/HBV/HCV, recent major surgery risks, drug abuse history, pregnancy/lactation intentions during the study period.

Inclusion Criteria

Contraceptive use consistent with local regulations, where available, for individuals participating in clinical studies. Women of childbearing potential must have a negative serum pregnancy test during screening and a negative urine pregnancy test at baseline before receiving investigational medicinal product (IMP)

Abnormal levels of at least 1 of the following enzymes: creatine kinase (CK), aldolase, lactate dehydrogenase, aspartate aminotransaminase (AST), alanine aminotransferase (ALT), based on central laboratory results

I was diagnosed with dermatomyositis before I turned 18, and it was within the last 5 years.

Exclusion Criteria

You have had an allergic reaction to the experimental medication or any of its ingredients.

You have another autoimmune disease that could make it difficult to accurately evaluate your symptoms of idiopathic inflammatory myopathy (IIM) or might put you at increased risk.

Positive serum test at screening for active viral infection with any of the following conditions: Hepatitis B virus (HBV); Hepatitis C virus (HCV); HIV

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 on top of background treatment

12 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
• PBO

Trial Overview The trial is testing Efgartigimod PH20 SC's effectiveness compared to a placebo in improving symptoms of IIM as measured by Total Improvement Score (TIS). It includes adults diagnosed with certain subtypes of IIM who will receive either the investigational medication or a placebo while continuing their standard treatment for IIM.

How Is the Trial Designed?

Group I: EFG PH20 SC Experimental Treatment 1 Intervention

participants receiving efgartigimod PH20 SC on top of background treatment

Group II: PBO PH20 SC Placebo Group 1 Intervention

participants receiving placebo PH20 SC on top of background treatment

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, 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]

In a study involving 251 patients, BYM338 (bimagrumab) did not significantly improve motor function, as measured by the 6-minute walking distance, compared to placebo after 52 weeks of treatment.

However, all BYM338 dose groups showed a dose-dependent increase in lean body mass, indicating a potential benefit in skeletal muscle mass despite the lack of improvement in motor function.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Late phase II/III study of BYM338 in patients with sporadic inclusion body myositis (RESILIENT): Japanese cohort data]. Mori-Yoshimura, M., Yamashita, S., Suzuki, N., et al.[2022]

Efgartigimod effectively reduces levels of pathogenic anti-HMGCR autoantibodies and total IgG in a humanized mouse model of immune-mediated necrotizing myopathies, demonstrating significant therapeutic potential.

In both preventive and therapeutic settings, efgartigimod not only prevents muscle fiber necrosis but also promotes muscle fiber regeneration, leading to restored muscle strength, which supports further clinical trials in patients with IMNM.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod restores muscle function in a humanized mouse model of immune-mediated necrotizing myopathy. Julien, S., van der Woning, B., De Ceuninck, L., et al.[2023]

In a study of 13 treatment-resistant patients with inflammatory myopathies, high-dose intravenous immunoglobulin (IVIG) treatment led to improved muscle function in only three patients and decreased creatinine kinase (CK) levels in five, indicating limited efficacy.

Despite some clinical improvements, the study found no significant changes in key immunological markers in muscle biopsies after IVIG treatment, suggesting that IVIG may not effectively modulate the immune response in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Limited effects of high-dose intravenous immunoglobulin (IVIG) treatment on molecular expression in muscle tissue of patients with inflammatory myopathies. Barbasso Helmers, S., Dastmalchi, M., Alexanderson, H., et al.[2022]

Intravenous immunoglobulin (IVIg) treatment significantly improved clinical scores in patients with Stiff-Person Syndrome (SPS) and dermatomyositis (DM) over 3 months, while scores returned to baseline when switched to placebo, indicating its efficacy in these conditions.

In addition to clinical improvements, IVIg treatment led to notable changes in tissue markers, including reduced inflammation and alterations in immunoregulatory genes, demonstrating its mechanism of action in restoring tissue health.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravenous immunoglobulin in patients with anti-GAD antibody-associated neurological diseases and patients with inflammatory myopathies: effects on clinicopathological features and immunoregulatory genes. Dalakas, MC.[2018]

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 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]

The study utilized artificial intelligence to evaluate treatment responses in patients with Inflammatory Idiopathic Myopathies (IIM) receiving intravenous immunoglobulin (IVIg) and subcutaneous immunoglobulin (20%SCIg), identifying key predictors of treatment success based on muscle strength and disease activity scores.

Results indicated that muscle strength improvement, as measured by the MMT8 score, was associated with the presence of dysphagia and skin disorders, suggesting that IVIg therapy may be more effective in patients with active systemic disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A machine learning analysis to predict the response to intravenous and subcutaneous immunoglobulin in inflammatory myopathies. A proposal for a future multi-omics approach in autoimmune diseases. Danieli, MG., Tonacci, A., Paladini, A., et al.[2022]

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]

Serum levels of B cell activating factor (BAFF) were significantly higher in patients with myositis compared to healthy controls, suggesting a potential role in the disease's pathology.

Higher BAFF levels were associated with specific autoantibodies, particularly anti-Jo-1, and correlated with muscle damage markers, indicating that BAFF may serve as a therapeutic target in myositis treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Increased serum levels of B cell activating factor (BAFF) in subsets of patients with idiopathic inflammatory myopathies. Krystufková, O., Vallerskog, T., Helmers, SB., et al.[2009]

In a double-blind, placebo-controlled study, high-dose intravenous immune globulin (IVIG) showed moderate to dramatic improvement in 75% of patients with dermatomyositis, indicating its efficacy as a treatment for this condition.

IVIG works by inhibiting harmful cytokines, blocking macrophage activity, and preventing damage to blood vessels in muscle tissue, which helps reduce inflammation in inflammatory myopathies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical benefits and immunopathological correlates of intravenous immune globulin in the treatment of inflammatory myopathies. Dalakas, MC.[2005]

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 ... The data presented at EULAR highlight efgartigimod's potential as a precision therapy for patients living with myositis and Sjogren's disease.

2. argenxmedical.com argenxmedical.com/content/dam/medical-affairs/congress-posters/nuerology/eular-june-2025/Efficacy%20and%20Safety%20of%20Efgartigimod%20PH20%20SC%20in%20Adult%20Participants%20With%20Active%20Idiopathic%20Inflammatory%20MyopathyPhase%202%20Results%20From%20the%20ALKIVIA%20Study.pdf

Efficacy and Safety of Efgartigimod PH20 SC in Adult ... Phase 2 Results From the ALKIVIA Study. Hector Chinoy,1,2 Sebastian C ... PH20; PM, polymyositis; R, randomization; SC, subcutaneous.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05523167

NCT05523167 | A Study to Investigate the Efficacy and ... This study's purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S0003496725010581

OP0002 EFFICACY AND SAFETY OF EFGARTIGIMOD ... Efgartigimod PH20 SC led to significant improvement over placebo in TIS and key secondary endpoints with good safety and tolerability.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT05979441

NCT05979441 | A Study to Assess the Long-term Safety ... The purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with IIM who previously ...

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

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, ...