Efgartigimod for Myasthenia Gravis

Overseen By François Henri Jacques, Doctor

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Clinique Neuro-Outaouais

Must be taking: IVIG

Must not be taking: Rituxan, Eculizumab

Disqualifiers: Hepatitis, HIV, TB, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: 26

Duration: 24 weeks

30 Participants Needed

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 requires that your myasthenia gravis treatment has been stable for the past four months, with no changes in certain medications like corticosteroids for the past three months.

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

Efgartigimod has been shown to significantly reduce disease burden 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 durable and consistent, and the drug was generally well tolerated with mostly mild to moderate side effects. ^{1 2 3 4 5}

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. ^{1 2 4 6 7}

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 generalized myasthenia gravis, especially those with specific antibodies. ^{1 2 3 4 5}

What is the purpose of this trial?

This study is an open label, single center, prospective, 26 weeks study with descriptive analysis where IVIG is replaced by efgartigimod therapy. MG-ADL and MGQOL evaluations will occur weekly throughout the study to week 26.

Eligibility Criteria

This trial is for patients with Myasthenia Gravis who rely on regular intravenous immunoglobulin (IVIG) therapy. Participants should be stable on their IVIG treatment before joining. Specific inclusion and exclusion criteria details are not provided, but typically these would outline health conditions and factors that qualify or disqualify someone from participating.

Inclusion Criteria

I have been getting IVIG treatments for myasthenia gravis for over a year.

No modification or addition of NSISTs in the past six months

I have Myasthenia Gravis confirmed by tests or improvement with specific medication.

Exclusion Criteria

I had my thymus gland removed within the last 3 months.

Patients that are pregnant or considering becoming pregnant in the next 6 months

I have not had rituxan, eculizumab, or plasma exchange in the last 6 months.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

1 week

Treatment

Efgartigimod is administered as a one-hour intravenous infusion every week for 4 infusions followed by a four-week break, repeated for a total of four treatment cycles

24 weeks

Weekly visits for infusions

Observation

Participants undergo a two-week observation period with an end of study visit

2 weeks

End of study visit at week 26

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Efgartigimod

Trial Overview The study is testing efgartigimod as a replacement therapy for IVIG in Myasthenia Gravis patients over a period of 26 weeks. It's an open-label trial, meaning both the researchers and participants know what treatment is being given, at a single center with weekly evaluations using MG-ADL and MGQOL scales.

Participant Groups

Group I: treatment Experimental Treatment 1 Intervention

Efgartigimod infusion

Who Is Running the Clinical Trial?

Clinique Neuro-Outaouais

Lead Sponsor

Trials

7

Recruited

240+

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

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. Bril, V., Howard, JF., Karam, C., et al.[2023]

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, 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, an FcRn antagonist, has been shown in clinical trials to effectively improve clinical status and reduce autoantibody levels in patients with myasthenia gravis, with no significant safety concerns reported.

This treatment has received regulatory approvals in the United States, Japan, and Europe, suggesting its potential effectiveness across various subgroups and severities of myasthenia gravis.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical efficacy and safety of efgartigimod for treatment of myasthenia gravis. Sivadasan, A., Bril, V.[2023]

References

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

Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. [2023]

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

Safety and outcomes with efgartigimod use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice. [2023]

3. 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]

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

Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. [2020]

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

Efgartigimod: First Approval. [2022]

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]

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

Clinical efficacy and safety of efgartigimod for treatment of myasthenia gravis. [2023]