CHU de Nice
Nice, Alpes-Maritimes, 06000, France
Location status: Recruiting
Location contact
Sabrina SACCONI, pu-ph
CONTACT
Sabrina SACCONI, pu-ph
CONTACT
NCT Number: NCT07072988
Generalized Myasthenia Gravis (gMG) is a rare chronic autoimmune disorder causing muscle weakness and fatigue, primarily due to autoantibodies that disrupt neuromuscular junction function. The most common antibodies target nicotinic acetylcholine receptors (AChR), with others such as anti-MuSK and anti-LRP4 being less prevalent. The conventional gMG treatments include acetylcholinesterase inhibitors, corticosteroids, immunosuppressant and, in case of myasthenic crisis, plasma exchange (PLEX) and intravenous immunoglobulins (IVIG). Treatment aims to achieve minimal manifestation status (MMS), but many patients face persistent symptoms or side effects. Corticosteroids, while effective, carry significant risks, especially for long-term use, such as, increased infection and cardiovascular risks, chronic conditions like hypertension, diabetes, and osteoporosis and quality of life impacts, including weight gain and mood changes. Elderly patients, who form the majority of the gMG population, are particularly vulnerable due to age-related comorbidities, which limit treatment options and prolong corticosteroid reliance. This contributes to increased mortality, disability, and dependency. Efgartigimod (EFG), a novel therapeutic targeting the neonatal Fc receptor (FcRn), accelerates degradation of pathogenic IgG antibodies, including anti-AChR. Clinical trials demonstrated its efficacy and safety in reducing antibody levels, improving muscle strength, and enhancing quality of life. Both intravenous (IV) and subcutaneous (SC) forms are effective and well tolerated. Approved in the United States and subsequently in Japan and Europe, EFG became available in France in 2023. The present multicenter observational study aims to evaluate the real-life impact of EFG in elderly gMG patients struggling with corticosteroid side effects or comorbidity exacerbations. The objectives of this study include the assessing EFG's ability to enable corticosteroid reduction and monitoring improvements in gMG symptoms, quality of life, comorbidities, and overall health.
This approach highlights a shift towards targeted therapies that balance efficacy with reduced treatment-related burdens for vulnerable gMG populations.
Interested in participating?
Request Info65 year and older
All sexes
Interventional
Phase 4
Nice, Alpes-Maritimes, 06000, France
Location status: Recruiting
Sabrina SACCONI, pu-ph
CONTACT
Sabrina SACCONI, pu-ph
CONTACT
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
Administration of efgartigimod, a neonatal Fc receptor (FcRn) antagonist, given either intravenously (IV) or subcutaneously (SC) to treat patients with generalized Anti-AChR Myasthenia Gravis.
Time frame: 12 mois
evaluate at 12 months the change from baseline in the mean dose of corticosteroids in gMG patients switched to EFG
Time frame: 12 months
change from baseline of the percentage of patient treated with <20 mg of corticosteroids
Time frame: 12 months
change from baseline of the percentage of patient treated with ≥20 mg - ≤50 mg of corticosteroids
Time frame: 12 months
change from baseline of the percentage of patient treated with >50 mg of corticosteroids
Time frame: 12 months
change from baseline in GTI
Contact information is provided by the study sponsor or research team.
Centre Hospitalier Universitaire de Nice
Other
A Multicenter, Prospective, Observational, Open-label Study to Evaluate the Benefit of Corticoid Sparing in Elderly With Generalized AntiRAch Myasthenia Gravis Treated With IV or SC Efgartigimod
Acronym: OPTIMAGE
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
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