Efgartigimod for Myasthenia Gravis
(ADAPT Jr SC Trial)
Recruiting in Palo Alto (17 mi)
+8 other locations
Age: < 18
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2 & 3
Recruiting
Sponsor: argenx
Must be taking: Corticosteroids, Immunosuppressants
Disqualifiers: Pregnancy, Infection, Autoimmune disease, Cancer, others
No Placebo Group
Prior Safety Data
Approved in 4 Jurisdictions
Trial Summary
What is the purpose of this trial?The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study.
The participants will be in the study for up to 14 weeks.
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 a stable dose of their current MG therapy for at least one month before screening. It's best to discuss your specific medications with the study team.
What data supports the effectiveness of the drug Efgartigimod for treating myasthenia gravis?
Efgartigimod has been shown in clinical trials to significantly reduce disease symptoms and improve muscle strength and quality of life in patients with generalized myasthenia gravis. It was well tolerated, with most side effects being mild to moderate, and has been approved for use in several countries.
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.
12456What makes the drug Efgartigimod PH20 SC unique for treating Myasthenia Gravis?
Efgartigimod PH20 SC is unique because it is designed to reduce the levels of antibodies that attack the body's own tissues, which is a novel approach for treating Myasthenia Gravis. This drug is administered subcutaneously (under the skin), offering a different route compared to some other treatments that may require intravenous administration.
7891011Eligibility Criteria
This trial is for children aged 2 to less than 18 with generalized Myasthenia Gravis (gMG), confirmed by tests. They should understand the study and follow its rules, have tried other gMG treatments without enough improvement, and be on a stable dose of current medications for at least a month. Participants must use birth control if applicable.Inclusion Criteria
I understand the study requirements and can consent to participate.
Participant agrees to use birth control consistent with local regulations and people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug
I've tried certain medications for my condition without much success but am on a stable treatment plan now.
+2 more
Exclusion Criteria
I had cancer but have been free of it for over 3 years or was treated successfully for certain cancers.
I have not received a live vaccine in the last 4 weeks.
Positive PCR test for SARS-CoV-2 at screening
+13 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive injections of efgartigimod PH20 SC and are monitored for safety, pharmacokinetics, and pharmacodynamics
12 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
2 weeks
Open-label extension (optional)
Eligible participants may roll over to an open-label extension study for continued treatment
Participant Groups
The study tests Efgartigimod PH20 SC in kids with gMG to find the right dose based on how their body absorbs and responds to it. It involves injections of this drug, monitoring over up to 14 weeks, followed by an optional extension phase for further observation.
1Treatment groups
Experimental Treatment
Group I: Efgartigimod PH20 SCExperimental Treatment1 Intervention
Participants aged 12 to \<18 years receiving efgartigimod PH20 SC treatment
Efgartigimod PH20 SC is already approved in European Union, United States, Japan, China for the following indications:
🇪🇺 Approved in European Union as VYVGART for:
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
🇺🇸 Approved in United States as VYVGART Hytrulo for:
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
- Chronic inflammatory demyelinating polyneuropathy (CIDP)
🇯🇵 Approved in Japan as VYVDURA for:
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
🇨🇳 Approved in China as Efgartigimod alfa injection (subcutaneous injection) for:
- Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Neurology Rare Disease CenterDenton, TX
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Who Is Running the Clinical Trial?
argenxLead 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.
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 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).
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.
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.
Cardiovascular safety profile of a fixed-dose combination of glycopyrrolate and formoterol fumarate delivered via metered dose inhaler using co-suspension delivery technology. [2019]Label="BACKGROUND">Glycopyrrolate/formoterol fumarate (GFF) metered dose inhaler (MDI) is a fixed-dose combination of the long-acting muscarinic antagonist (LAMA), glycopyrrolate (GP), and the long-acting β2-agonist (LABA), formoterol fumarate (FF), delivered via metered dose inhaler using innovative co-suspension delivery technology. Here we report the results of two studies that examined the cardiovascular safety of GFF MDI.
Pharmacokinetics of multiple inhaled NVA237 doses in patients with chronic obstructive pulmonary disease (COPD). [2019]NVA237 (glycopyrronium bromide) is a once-daily longacting muscarinic antagonist (LAMA) in development for the treatment of chronic obstructive pulmonary disease (COPD). This study investigated the pharmacokinetics (PK) of NVA237 following single and repeated once-daily inhalation in mild-tomoderate COPD patients.
A randomized, seven-day study to assess the efficacy and safety of a glycopyrrolate/formoterol fumarate fixed-dose combination metered dose inhaler using novel Co-Suspension™ Delivery Technology in patients with moderate-to-very severe chronic obstructive pulmonary disease. [2022]Label="BACKGROUND">Long-acting muscarinic antagonist/long-acting β2-agonist combinations are recommended for patients whose chronic obstructive pulmonary disease (COPD) is not managed with monotherapy. We assessed the efficacy and safety of glycopyrrolate (GP)/formoterol fumarate (FF) fixed-dose combination delivered via a Co-Suspension™ Delivery Technology-based metered dose inhaler (MDI) (GFF MDI).
Delivery characteristics of a low-resistance dry-powder inhaler used to deliver the long-acting muscarinic antagonist glycopyrronium. [2023]The long-acting muscarinic antagonist (LAMA) glycopyrronium (NVA237) has recently been approved as a once-daily treatment for COPD. The objectives of this study were to determine the dose delivery characteristics of glycopyrronium and compare them with those of the LAMA tiotropium, both delivered by their respective capsule-based dry-powder inhalers (DPIs).
Efficacy and safety of NVA237 versus placebo and tiotropium in patients with COPD: the GLOW2 study. [2022]NVA237 (glycopyrronium bromide) is a once-daily long-acting muscarinic antagonist (LAMA) in development for chronic obstructive pulmonary disease (COPD). The GLycopyrronium bromide in COPD airWays clinical Study 2 (GLOW2) evaluated the efficacy and safety of NVA237 in moderate-to-severe COPD over 52 weeks. Patients were randomised 2:1:1 to NVA237 50 μg, placebo or open-label tiotropium 18 μg for 52 weeks. Primary end-point was trough forced expiratory volume in 1 s (FEV(1)) at 12 weeks. 1,066 patients were randomised, 810 completed the study. At week 12, trough FEV(1) increased significantly by 97 mL with NVA237 (95% CI 64.6-130.2; p