20 Participants Needed

Efgartigimod for Myasthenia Gravis

AA
Overseen ByAlyssa Avilez, BS
Age: 18+
Sex: Any
Trial Phase: Phase 4
Sponsor: University of Colorado, Denver
Must be taking: Cholinesterase inhibitors
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

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 excludes those who have used certain treatments like IVIG, plasma exchange, or efgartigimod recently. It also excludes those using specific medications like ravulizumab or eculizumab.

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

Efgartigimod has been shown to significantly reduce disease symptoms and improve muscle strength and quality of life in patients with generalized myasthenia gravis, as demonstrated in the phase 3 ADAPT trial. The benefits were consistent and well-tolerated, with most side effects being mild to moderate.12345

Is efgartigimod safe for humans?

Efgartigimod has been generally well tolerated in clinical trials for myasthenia gravis, with most side effects being mild to moderate. It has been approved for use in several countries, indicating a favorable safety profile.12467

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. This mechanism helps improve muscle strength and quality of life for patients with myasthenia gravis, and it is administered intravenously.12345

What is the purpose of this trial?

This study plans to learn more about if the drug efgartigimod can be used in the hospital to treat exacerbations in participants with myasthenia gravis (MG). Efgartigimod has been approved by the FDA for ongoing (chronic) treatment of generalized MG in adult patients who are anti-acetylcholine receptor (AChR) antibody positive but has not been studied in the treatment of worsening weakness requiring hospital admission (known as "exacerbation"). This investigation aims to see if using efgartigimod in this way improves symptoms and recovery from exacerbation, and how it affects certain MG markers in the blood. The main questions it aims to answer are:* Is efgartigimod effective as a hospital-administered acute therapy for participants with worsening MG (MG exacerbation) who require hospitalization?* Will efgartigimod lead to clinical improvement with a similar reduction in validated research scales, such as the Quantitative MG (QMG) scale, as standard of care therapies?Participants will receive 4 doses of efgartigimod over the course of 4 weeks with an additional follow-up visit at the clinic.

Research Team

TR

Thomas Ragole, MD

Principal Investigator

University of Colorado, Denver

Eligibility Criteria

This trial is for hospitalized adults with myasthenia gravis (MG) experiencing a worsening of symptoms, known as an exacerbation. Participants must have MG that's confirmed by specific antibodies. Details on who can't join are not provided.

Inclusion Criteria

Abnormal single fiber EMG
Abnormal decrement on slow repetitive nerve stimulation testing
Quantitative Myasthenia Gravis (QMG) scale ≥ 11
See 6 more

Exclusion Criteria

I am currently using ravulizumab or eculizumab.
Intubation prior to ability to sign informed consent or intubation within 24 hours of hospitalization
I don't have health issues that could affect study assessments.
See 6 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive 4 doses of efgartigimod via intravenous (IV) infusion on days 1, 4, 11, and 18

4 weeks
4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with assessments at day 32

2 weeks
1 visit (in-person)

Treatment Details

Interventions

  • Efgartigimod
Trial Overview The study tests if efgartigimod, already approved for chronic MG treatment, is effective when given in the hospital to treat acute MG exacerbations. Patients will receive four doses over four weeks and be monitored using the QMG scale.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: EfgartigimodExperimental Treatment1 Intervention
Participants receive 4 doses of efgartigimod via intravenous (IV) infusion over the course of the study on days 1, 4, 11 and 18.

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Colorado, Denver

Lead Sponsor

Trials
1,842
Recruited
3,028,000+

argenx

Industry 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

Findings from Research

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.
Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use.Heo, YA.[2023]
In a study of 37 patients with acetylcholine receptor antibody-positive generalized myasthenia gravis, 72% experienced a significant improvement in daily living activities after one cycle of efgartigimod, indicating its efficacy in managing symptoms.
Efgartigimod treatment resulted in a substantial reduction in IgG levels and was generally well-tolerated, with only mild side effects reported, although one patient had to discontinue due to a Clostridium difficile infection.
Safety and outcomes with efgartigimod use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice.Katyal, N., Halldorsdottir, K., Govindarajan, R., et al.[2023]
Efgartigimod, an engineered antibody, significantly improves muscle function and strength in patients with generalized myasthenia gravis (gMG), as shown by notable enhancements in Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) scores over a 10-week period.
Improvements were observed across all muscle function subdomains (bulbar, ocular, limb/gross motor, and respiratory) within 1 to 2 weeks after the first infusion, correlating with reduced levels of pathogenic IgG, indicating its mechanism of action in decreasing autoantibody levels.
Effect of efgartigimod on muscle group subdomains in participants with generalized myasthenia gravis: post hoc analyses of the phase 3 pivotal ADAPT study.Bril, V., Howard, JF., Karam, C., et al.[2023]

References

Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. [2023]
Safety and outcomes with efgartigimod use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice. [2023]
Effect of efgartigimod on muscle group subdomains in participants with generalized myasthenia gravis: post hoc analyses of the phase 3 pivotal ADAPT study. [2023]
Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. [2020]
Efgartigimod: First Approval. [2022]
Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. [2022]
Clinical efficacy and safety of efgartigimod for treatment of myasthenia gravis. [2023]
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