Efgartigimod for Myasthenia Gravis

(ADAPT SERON Trial)

Recruiting in Palo Alto (17 mi)

+88 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: argenx

Must be taking: Acetylcholinesterase inhibitors, Steroids

Disqualifiers: Autoimmune disease, Cancer, HIV, others

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 4 Jurisdictions

Trial Summary

What is the purpose of this trial?

The primary purpose of this study is to measure the efficacy and safety of efgartigimod intravenously (IV) compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG). Other objectives are to assess long-term efficacy, safety, and tolerability of efgartigimod. Study will consist of: * Screening * Part A: participants will be randomized to receive either efgartigimod IV or placebo * Part B: participants completing part A will receive open-label efgartigimod IV

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it mentions that participants should be on a stable dose of their current Myasthenia Gravis therapy before screening. It's best to discuss your specific medications with the trial team.

What data supports the effectiveness of the drug Efgartigimod for treating Myasthenia Gravis?

Efgartigimod has been shown in clinical trials to significantly reduce disease symptoms and improve muscle strength and quality of life in patients with generalized myasthenia gravis. It was well tolerated, with most side effects being mild to moderate, and has been approved for use in several countries.

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Is efgartigimod safe for humans?

Efgartigimod, also known as Vyvgart, has been generally well tolerated in clinical trials for myasthenia gravis, with most side effects being mild to moderate.

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How is the drug efgartigimod different from other treatments for myasthenia gravis?

Efgartigimod is unique because it is the first drug that works by blocking the neonatal Fc receptor, which reduces harmful antibodies in the body, helping to improve muscle strength and quality of life for people with myasthenia gravis. It is administered intravenously and has shown rapid and durable benefits in clinical trials.

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Eligibility Criteria

This trial is for adults with seronegative Generalized Myasthenia Gravis (gMG), who can consent and follow the study plan. They must use birth control if applicable, have a negative pregnancy test if female, and show signs of MG that improve with certain treatments or tests.

Inclusion Criteria

I can sign the consent form and follow the study rules.

The participant agrees to use contraceptive measures consistent with local regulations and the women of child-bearing potential (WOCBP) must have a negative serum pregnancy test result at screening and a negative urine pregnancy test result at baseline before receiving the study drug

I am of legal age to consent to join a study.

Exclusion Criteria

I had cancer but have been free of it for over 3 years, or I had specific non-aggressive cancers treated successfully.

I have used certain medications as outlined in the trial protocol.

Known hypersensitivity to study drug or one of its excipients (inactive ingredients)

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part A: Treatment

Participants are randomized to receive either efgartigimod IV or placebo

4 weeks

Weekly visits (in-person)

Part B: Open-label Treatment

Participants completing Part A will receive open-label efgartigimod IV

4 weeks

Weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Participant Groups

The study compares Efgartigimod IV to a placebo in treating gMG. Participants are randomly chosen to receive either the drug or placebo. Those completing this part will later all receive Efgartigimod in an open-label extension.

Group I: Efgartigimod IV Experimental Treatment 1 Intervention

Patients receiving efgartigimod IV in both part A and part B

Group II: Placebo Placebo Group 2 Interventions

Patients receiving placebo during part A and receiving efgartigimod IV during part B

Efgartigimod is already approved in European Union, United States, Canada, Japan for the following indications:

🇪🇺 Approved in European Union as Vyvgart for:

• Generalized Myasthenia Gravis (gMG)

🇺🇸 Approved in United States as Vyvgart for:

• Generalized Myasthenia Gravis (gMG)
• Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

🇨🇦 Approved in Canada as Vyvgart for:

• Generalized Myasthenia Gravis (gMG)

🇯🇵 Approved in Japan as Vyvgart for:

• Generalized Myasthenia Gravis (gMG)

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Northwestern Medicine - Northwestern Memorial Hospital Chicago, IL

First Choice Neurology Boca Raton Boca Raton, FL

Henry Ford Health - Henry Ford Hospital Detroit, MI

Medsol Clinical Research Center Inc Port Charlotte, FL

More Trial Locations

Who Is Running the Clinical Trial?

argenx Lead Sponsor

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. [2023] Intravenous efgartigimod alfa (also known as efgartigimod alfa-fcab in the USA; Vyvgart®) is the first neonatal Fc receptor antagonist approved in several countries worldwide, including the USA and EU for the treatment of generalised myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody positive, and in Japan for the treatment of gMG regardless of antibody status. In the double-blind, placebo-controlled phase 3 ADAPT trial in patients with gMG, efgartigimod alfa significantly and rapidly reduced disease burden and improved muscle strength and quality of life compared with placebo. The clinical benefits of efgartigimod alfa were durable and reproducible. Furthermore, in an interim analysis of the ongoing open-label phase 3 ADAPT+ extension trial, efgartigimod alfa provided consistent clinically meaningful improvements in patients with gMG. Efgartigimod alfa was generally well tolerated, with most adverse events being mild to moderate in severity.

2. New Zealand pubmed.ncbi.nlm.nih.gov

Efgartigimod: First Approval. [2022] Efgartigimod (efgartigimod alfa-fcab, Vyvgart™) is a first-in-class neonatal Fc receptor antagonist being developed by argenx for the treatment of autoimmune diseases including myasthenia gravis. In December 2021, intravenous efgartigimod received its first approval in the USA for the treatment of generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive. Intravenous efgartigimod has also been evaluated for generalized myasthenia gravis in various other countries, with the agent subsequently approved in Japan in January 2022 for generalized myasthenia gravis patients regardless of antibody status and in preregistration stage in the EU. Several clinical studies of intravenous and subcutaneous formulation of efgartigimod are also being investigated for other autoimmune diseases including bullous pemphigoid, chronic inflammatory demyelinating polyradiculoneuropathy, immune thrombocytopenia, autoimmune myositis and pemphigus. This article summarizes the milestones in the development of efgartigimod leading to this first approval for generalized myasthenia gravis.

3. United States pubmed.ncbi.nlm.nih.gov

Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. [2020] To investigate safety and explore efficacy of efgartigimod (ARGX-113), an anti-neonatal Fc receptor immunoglobulin G1 Fc fragment, in patients with generalized myasthenia gravis (gMG) with a history of anti-acetylcholine receptor (AChR) autoantibodies, who were on stable standard-of-care myasthenia gravis (MG) treatment.

4. United States pubmed.ncbi.nlm.nih.gov

Safety and outcomes with efgartigimod use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice. [2023] Multiple novel therapies have been approved for patients with myasthenia gravis. Our aim is to describe the early experience of efgartigimod use in acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR+ve gMG).

5. England pubmed.ncbi.nlm.nih.gov

Clinical efficacy and safety of efgartigimod for treatment of myasthenia gravis. [2023] Treatment of acute exacerbations and refractory myasthenia gravis (MG) remains challenging despite advances in immunotherapy. Frequent use of plasmapheresis and immunoglobulins are associated with adverse events and strain on resources. The neonatal Fc receptor (FcRn) facilitates IgG recycling and FcRn antagonism enhances the degradation of IgG pathogenic autoantibodies without compromising adaptive and innate immunity. Efgartigimod, an FcRN antagonist, has been shown in well-designed clinical trials to improve clinical status and reduce autoantibody levels without significant safety concerns. Efgartigimod has received approvals for use in the United States, Japan and Europe. It is plausible that efgartigimod is effective across different subgroups and varied spectrums of MG severity. Novel strategies involving FcRn modulation and long-term follow-up studies will help provide further insights and expand the therapeutic repertoire.

6. England 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. [2022] There is an unmet need for treatment options for generalised myasthenia gravis that are effective, targeted, well tolerated, and can be used in a broad population of patients. We aimed to assess the safety and efficacy of efgartigimod (ARGX-113), a human IgG1 antibody Fc fragment engineered to reduce pathogenic IgG autoantibody levels, in patients with generalised myasthenia gravis.

7. England pubmed.ncbi.nlm.nih.gov

Effect of efgartigimod on muscle group subdomains in participants with generalized myasthenia gravis: post hoc analyses of the phase 3 pivotal ADAPT study. [2023] Generalized myasthenia gravis (gMG) is a rare, chronic, neuromuscular autoimmune disease mediated by pathogenic immunoglobulin G (IgG) autoantibodies. Patients with gMG experience debilitating muscle weakness, resulting in impaired mobility, speech, swallowing, vision and respiratory function. Efgartigimod is a human IgG1 antibody Fc fragment engineered for increased binding affinity to neonatal Fc receptor. The neonatal Fc receptor blockade by efgartigimod competitively inhibits endogenous IgG binding, leading to decreased IgG recycling and increased degradation resulting in lower IgG concentration.