Observational Study on the Effects of Efgartigimod Alfa in Adults with Generalized Myasthenia Gravis in Italy

What this Trial Is About

Researchers are studying adult patients with generalized myasthenia gravis gMG who are positive for acetylcholine receptor AChR antibodies. The study aims to gather real-world evidence from Italian clinical practice on how efgartigimod alfa affects clinical outcomes and patient-reported experiences in those treated for gMG. This observational study will follow patients over a total duration of up to 23 months. The study involves collecting primary data at a baseline visit and follow-up visits at 6 and 12 months after baseline. Researchers will also gather secondary data from medical charts covering the 12 months before baseline, including details on MG-related therapies, exacerbations, hospitalizations, and the patients medical history. The treatment with efgartigimod alfa is decided independently by the treating physician as part of routine care, not influenced by study participation. Participants will be monitored through clinical assessments and questionnaires measuring changes in MG-Activities of Daily Living MG-ADL scores and the percentage achieving minimal symptom expression MSE up to 12 months. Additional measures include quality of life, fatigue, anxiety and depression, sleep quality, and caregiver burden scores tracked over the same period. The study collects comprehensive data to understand disease experience and treatment impact, with safety and outcomes evaluated throughout the 23-month period.

A Study to Evaluate the Impact of Efgartigimod on Overall Disease Experience of People Suffering From Generalized Myasthenia Gravis (gMG) in Italy