Evaluate the Benefit of Corticoid Sparing in Elderly With Generalized AntiRAch Myasthenia Gravis Treated With IV or SC Efgartigimod

Sponsor
Centre Hospitalier Universitaire de Nice
Study ID
NCT07072988
Phase
PHASE4
Status
Recruiting
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Conditions

  • Myasthenia Gravis, Generalized

Eligibility Criteria

Sex
ALL
Age
65 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Efgartigimod — DRUG
    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.

Study Details

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.

Key Dates

Start date
Sep 19, 2025
Status verified
Sep 2025
Primary completion
Oct 1, 2027
Completion
Oct 1, 2027

Study Design

Enrollment
50 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION

Arms

  • Experimental: Elderly with Generalized Anti-AChR Myasthenia Gravis Treated with IV or SC Efgartigimod

Primary Outcome Measure

evaluate at 12 months the change from baseline in the mean dose of corticosteroids in gMG patients switched to EFG [ Time Frame: 12 mois ]

Central Contacts

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