Efgartigimod IV for Myasthenia Gravis
(ADAPT Jr Trial)
Recruiting in Palo Alto (17 mi)
+84 other locations
Age: < 65
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2 & 3
Recruiting
Sponsor: argenx
Must be taking: Immunosuppressants, Steroids, AChE inhibitors
Must not be taking: Monoclonal antibodies, IVIg, PLEX
Disqualifiers: MGFA class I, IVb, V, others
No Placebo Group
Prior Safety Data
Approved in 3 Jurisdictions
Trial Summary
What is the purpose of this trial?
The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. Trial details include: * The maximum trial duration for each individual participant will be approximately 28 weeks * The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)
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 stable gMG therapy before screening. It's best to discuss your current medications with the trial team to understand any specific requirements.
What data supports the effectiveness of the drug Efgartigimod IV for treating myasthenia gravis?
Is Efgartigimod IV safe for humans?
How is the drug Efgartigimod IV different from other treatments for myasthenia gravis?
Efgartigimod IV is unique because it is the first drug that works by blocking the neonatal Fc receptor, which reduces harmful antibodies in the body, specifically for treating generalized myasthenia gravis. This mechanism is different from other treatments and has shown to quickly improve muscle strength and quality of life in patients.23467
Eligibility Criteria
This trial is for children and adolescents aged 2 to less than 18 with Generalized Myasthenia Gravis (gMG). They must have tried other treatments without success, not be pregnant, use contraception consistently, and not have had recent major surgery or certain infections. Those with mild gMG or severe muscle weakness from other causes can't join.Inclusion Criteria
I agree not to donate sperm during the trial.
I have been diagnosed with Generalized Myasthenia Gravis.
Female participants must have a negative pregnancy test.
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Exclusion Criteria
I have had a major surgery or serious disease recently.
I have had, or will have, a thymectomy within 3 months of joining the trial.
You are allergic to efgartigimod or any of its ingredients.
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Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment Part A
Participants receive efgartigimod IV for dose-confirmatory purposes
8 weeks
Treatment Part B
Participants continue receiving efgartigimod IV for treatment response-confirmatory purposes
18 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
2 weeks
Treatment Details
Interventions
- Efgartigimod IV (Monoclonal Antibodies)
Trial OverviewThe study tests Efgartigimod IV in young patients with gMG over approximately 28 weeks. It has two parts: an initial dose-confirmatory phase lasting 8 weeks and a subsequent treatment response-confirmatory phase of 18 weeks to assess the drug's effects on their condition.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: EfgartigimodExperimental Treatment1 Intervention
Patients receiving efgartigimod intravenous (IV) treatment
Efgartigimod IV is already approved in United States, European Union, United States 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)
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Investigator site 18 - US0010142Charlottesville, VA
Ann and Robert H Lurie Children's Hospital of Chicago - Main HospitalChicago, IL
University of Virginia (UVA) Health - Developmental Pediatrics ClinicCharlottesville, VA
Investigator Site 16 - US0010120Chicago, IL
More Trial Locations
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Who Is Running the Clinical Trial?
argenxLead Sponsor
References
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.
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.
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.
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.
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.
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.
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).