Generalized Myasthenia Gravis Generalized Myasthenia Gravis, Minimal Manifestation, Efgartigimod
Conditions
Interventions
Maintenance treatment with efgartigimod (intravenous infusion or subcutaneous injection)
L04AA58
Efgartigimod, FcRn inhibitor
Sponsors
Fujimaki Motoki
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Eligible patients must meet all of the following criteria. 1) Patients diagnosed with generalized myasthenia gravis according to the revised 2022 diagnostic criteria. 2) Patients who have been treated with efgartigimod (Vyvgart/Vyvgart Hytrulo) and who have achieved one of the following Minimal Manifestation (MM) states as the best score during the most recent three treatment cycles: - MG-ADL score = 18 years and < 80 years at the time of informed consent. 5) For patients who select subcutaneous self-administration, those who are judged by the investigator to be capable of acquiring appropriate injection techniques and reliably performing self-administration under self-management. 6) Patients who can continue the selected route of administration (Vyvgart: intravenous infusion; Vyvgart Hytrulo: subcutaneous injection) without change throughout the study period and who are able to attend scheduled study visits continuously from study initiation to study completion according to the prescribed schedule. 7) Patients who have received sufficient explanation regarding participation in this study, have fully understood the content, and have provided written informed consent based on their own free will.
Exclusion criteria
Exclusion criteria: Patients meeting any of the following criteria will be excluded from this study. 1) Patients currently receiving treatment with other targeted therapies, including FcRn inhibitors, complement inhibitors, or B-cell-depleting agents. 2) Patients who are carriers of hepatitis viruses or who have active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection. 3) Patients with positive nucleic acid quantification tests for HBV or HCV. 4) Patients with severe hepatic disease, defined as aspartate aminotransferase (AST/GOT) or alanine aminotransferase (ALT/GPT) >= 100 U/L. 5) Patients with severe renal disease. 6) Patients with active tuberculosis. 7) Patients with a current or past history of malignant neoplasms (excluding thymoma), except for those who have remained recurrence-free for at least 5 years. 8) Patients with suspected active infection. 9) Pregnant women or women who may be pregnant. 10) Breastfeeding women. 11) Patients who have received a live vaccine within 30 days prior to enrollment. 12) Patients who are otherwise judged by the principal investigator or sub-investigator to be unsuitable for participation i
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The proportion of subjects who maintained Minimal Manifestation (MM) status throughout the entire observation period, from initiation of efgartigimod treatment through the end of the 4-week post-treatment follow-up period, while continuing maintenance therapy with a prednisolone-equivalent dose of <= 5 mg/day under efgartigimod treatment. MM is defined as meeting either of the following criteria: MG-ADL score <= 2 MG Composite score <= 4 MM status is determined based on the best MG-ADL score observed during each treatment cycle, together with the physician's assessment at each study visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Changes in MG-related scores. a) MG-ADL score. b) MG Composite score. c) MG-QOL15 score. d) QMG (Quantitative Myasthenia Gravis) score. 2) Patient-reported outcomes (PROs). a) Satisfaction with treatment. b) Convenience and preference compared with previous treatments (assessed by questionnaire). 3) Changes in serum IgG levels. 4) Changes in and distribution of efgartigimod cycle intervals (dosing intervals). 5) Changes in anti-acetylcholine receptor (AChR) antibody and anti-MuSK antibody titers (measured at baseline, at the end of the study, and at the first blood sampling of each treatment cycle). 6) Time from enrollment to treatment discontinuation in patients who discontinued study treatment due to difficulty in maintaining MM status according to the criteria defined in Section 7.1. | — |
Contacts
Public ContactMotoki Fujimaki
University of Tsukuba Hospital
Outcome results
None listed