Efgartigimod for Myasthenia Gravis
Recruiting in Palo Alto (17 mi)
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Clinique Neuro-Outaouais
Must be taking: IVIG
Must not be taking: Rituxan, Eculizumab
Disqualifiers: Hepatitis, HIV, TB, Pregnancy, others
No Placebo Group
Pivotal Trial (Near Approval)
Prior Safety Data
Trial Summary
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.
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.
12345Is 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.
12467How 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.
12345Eligibility 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 am between 18 and 80 years old.
+4 more
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.
+4 more
Trial 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
Participant Groups
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.
1Treatment groups
Experimental Treatment
Group I: treatmentExperimental Treatment1 Intervention
Efgartigimod infusion
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Clinique Neuro-OutaouaisGatineau, Canada
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Who Is Running the Clinical Trial?
Clinique Neuro-OutaouaisLead Sponsor
argenxIndustry Sponsor
References
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.
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).
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.
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.
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.
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.
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.