What side effects are associated with this type of intervention?

Common treatments for Polymyositis include intravenous immunoglobulin (IVIg), rituximab, and mexiletine. IVIg can cause mild regional benefits but may also lead to adverse effects such as infections. Rituximab has been associated with serious adverse effects, including infections like pneumonia and cellulitis, as well as infusion reactions. Mexiletine, used for muscle stiffness, is generally well tolerated but can cause gastrointestinal discomfort and cardiac issues. These treatments aim to improve muscle strength and quality of life but come with potential side effects that should be discussed with a healthcare provider.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments specifically targeting FcRn inhibition for Polymyositis. Traditional treatments for Polymyositis include corticosteroids, immunosuppressants like methotrexate and azathioprine, and intravenous immunoglobulin (IVIG). Mycophenolate mofetil has also been used as a second-line agent in resistant cases. These treatments aim to reduce inflammation and modulate the immune response.

What other types of research exist?

Promising novel alternatives to Efgartigimod PH20 SC for Polymyositis patients include Rituximab, which has shown efficacy in various autoimmune conditions, and Mycophenolate Mofetil, which is used as an immunosuppressant in cases where other treatments are not tolerated. Additionally, intravenous immunoglobulin (IVIG) remains a viable option for its immunomodulatory effects. Patients should discuss these options with their healthcare provider to determine the best course of action based on their specific condition and treatment response.

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Monoclonal Antibodies

Efgartigimod for Myositis (ALKIVIA Trial)

Phase 2 & 3

Recruiting

Research Sponsored by argenx

Eligibility Criteria Checklist

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

Must have

Ability to consent in the jurisdiction in which the study is taking place and capable of giving signed informed consent.

A definite or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM)

Must not have

A clinically significant active infection at screening

Severe muscle damage

Timeline

Screening 3 weeks

Treatment Varies

Follow Up phase 2: 24 weeks; phase 3: 52 weeks

Summary

This trial is testing an injectable medication called efgartigimod PH20 SC in people with certain muscle inflammation diseases. The goal is to see if it helps improve their condition by lowering harmful proteins in their blood. The study focuses on patients with specific subtypes of these diseases who often don't respond well to usual treatments. Efgartigimod was developed for autoimmune diseases and has been approved for treating a specific muscle condition in adults.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not explicitly listed but may include reactions at the injection site due to Efgartigimod PH20 SC or general side effects associated with immunotherapy such as fatigue, headache, nausea or potential allergic reactions.

Eligibility Criteria

Inclusion Criteria

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

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I can legally consent and am able to sign the consent form.

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I have been diagnosed with idiopathic inflammatory myopathy.

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I have been diagnosed with polymyositis or antisynthetase syndrome.

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I have been diagnosed with immune-mediated necrotizing myopathy.

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I have been diagnosed with idiopathic inflammatory myopathy.

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I have been diagnosed with DM, JDM, PM (including ASyS), or IMNM.

Exclusion Criteria

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

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I do not have any serious infections currently.

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I have severe muscle damage.

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I have taken part in an efgartigimod study and received at least one dose of the trial medication.

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I have severe kidney problems.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ phase 2: 24 weeks; phase 3: 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~phase 2: 24 weeks; phase 3: 52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and phase 2: 24 weeks; phase 3: 52 weeks for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Total improvement score (TIS); measured on a [0,100] scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).

Secondary study objectives

Change in Patient Global Assessment of Disease Activity (PGA)

Change in Physician Global Assessment of Disease Activity (MDGA)

Change in manual muscle testing-8 (MMT8) score

+5 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: EFG PH20 SCExperimental Treatment1 Intervention

participants receiving efgartigimod PH20 SC on top of background treatment

Group II: PBO PH20 SCPlacebo Group1 Intervention

participants receiving placebo PH20 SC on top of background treatment

0 / 10Eligibility 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

Polymyositis (PM) is commonly treated with immunosuppressive agents such as corticosteroids (e.g., prednisolone), which reduce inflammation by inhibiting various inflammatory pathways. Other treatments include immunosuppressants like methotrexate and azathioprine, which inhibit the proliferation of immune cells, and intravenous immunoglobulin (IVIG), which modulates the immune response. Efgartigimod PH20 SC, an FcRn inhibitor, works by reducing the levels of pathogenic IgG antibodies, thereby decreasing immune-mediated muscle damage. These treatments are crucial for PM patients as they help to control muscle inflammation, prevent muscle weakness, and improve overall muscle function, thereby enhancing the quality of life.

Different mechanisms mediate structural changes and intracellular enzyme efflux following damage to skeletal muscle.Amelioration of inflammatory myopathies by glucagon-like peptide-1 receptor agonist via suppressing muscle fibre necroptosis.Forkhead Box O Signaling Pathway in Skeletal Muscle Atrophy.