Actively Recruiting

Phase 4
Age: 65Years +
All Genders
NCT07072988

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

Led by Centre Hospitalier Universitaire de Nice · Updated on 2025-09-24

50

Participants Needed

1

Research Sites

106 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 65Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Male or female subjects aged 6565 years
  • Diagnosed with generalized Myasthenia Gravis with confirmed antibody positivity for AChR
  • Meet MG Foundation of America clinical criteria for generalized MG class IIa, IIb, IIIa, or IIIb
  • Suitable for efgartigimod treatment
  • MG-ADL score of 2 or higher
  • Receiving stable intermediate (20-50 mg) or high (>50 mg) daily oral corticosteroids for at least 3 months
  • Developed or worsened comorbidities after corticosteroid use including type 2 diabetes, hypertension, overweight, sleep disorders, or irritability
  • Able to understand and provide written informed consent
  • Willing and able to comply with study visits, treatment, tests, and contraceptive guidelines
  • Affiliated with a European social security system
Not Eligible

You will not qualify if you...

  • Current mental condition affecting study compliance or understanding, including MMSE score below 28
  • Any illness or condition that could confound study results or increase treatment risk
  • Active or uncontrolled infections
  • Ongoing malignancy except certain skin or cervical cancers fully treated
  • Vaccinated with live or live-attenuated vaccines within 6 weeks
  • Recent treatment with anti-complement, anti-FcRn, anti-CD20 therapies, or plasma exchange within specified washout periods
  • End-stage disease or conditions preventing evaluation or treatment
  • Legal protection status preventing study participation according to French law

AI-Screening

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Trial Site Locations

Total: 1 location

1

CHU de Nice

Nice, Alpes-Maritimes, France, 06000

Actively Recruiting

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Research Team

S

Sabrina SACCONI, pu-ph

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

PREVENTION

Number of Arms

1

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