Long-term Safety of Efgartigimod for Myasthenia Gravis

(ADAPT Jr + Trial)

Recruiting at 43 trial locations

Overseen By Sabine s Coppieters, MD

Age: < 65

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: argenx

Disqualifiers: Pregnancy, Lactation, Autoimmune disease, others

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

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

12 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing the long-term safety of two forms of a medication called efgartigimod, given either through a drip or as an injection. It is aimed at patients with generalized Myasthenia Gravis, a condition that causes muscle weakness. The medication works by lowering harmful proteins in the blood that attack muscles.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

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

Research shows that Efgartigimod significantly reduces disease symptoms and improves muscle strength and quality of life in patients with generalized myasthenia gravis, as demonstrated in the phase 3 ADAPT trial. The drug was well tolerated, with most side effects being mild to moderate. ^{1 2 3 4 5}

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

Efgartigimod is unique because it is the first drug that targets the neonatal Fc receptor to reduce harmful antibodies in the body, offering a new approach for treating myasthenia gravis. It is administered intravenously and has shown rapid and significant improvements in muscle strength and quality of life for patients. ^{1 2 3 4 6}

Eligibility Criteria

This trial is for children who have a muscle weakness condition called generalized myasthenia gravis and were part of an earlier study (ARGX-113-2006). They must not plan to donate sperm or become pregnant, agree to use contraception, and understand the trial's requirements. Those with hypersensitivity to efgartigimod, recent live vaccines, other autoimmune diseases, or uncontrolled infections cannot join.

Inclusion Criteria

Participants must have completed ARGX-113-2006 or ARGX-113-2207 and agreed to participate in the ARGX-113-2008 trial

I am a female of childbearing age and my pregnancy test before the trial was negative.

Participants who qualify for retreatment in trial ARGX-113-2006 but cannot complete the required visits within the trial's timeframe

Exclusion Criteria

Known hypersensitivity reaction to efgartigimod or any of its excipients

Early discontinuation from ARGX-113-2006 or ARGX-113-2207 treatment

I do not have an autoimmune disease or any condition that could affect my gMG symptoms or increase my risk.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Efgartigimod IV or Efgartigimod PH20 SC treatment

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Efgartigimod IV

Trial Overview The trial is testing the long-term safety of a medication called Efgartigimod IV in children who previously participated in another related study. It aims to ensure that this treatment continues to be safe over an extended period.

Participant Groups

Group I: Efgartigimod or Efgartigimod PH20 SC Experimental Treatment 1 Intervention

Patients receiving Efgartigimod IV treatment or Efgartigimod PH 20 SC treatment

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

Approved in United States as Vyvgart for:

• Generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive

Approved in European Union as Vyvgart for:

• Generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive

Approved in United States as Vyvgart Hytrulo for:

• Generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive
• Chronic inflammatory demyelinating polyneuropathy (CIDP)

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

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]

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]

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]

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

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

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

Efgartigimod: First Approval. [2022]

4. 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]

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

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

6. Germany pubmed.ncbi.nlm.nih.gov

Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT). [2023]