NIPH Clinical Trials Search

Effect of efgartigimod on maintaining stable symptom status

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Generalized Myasthenia Gravis
Date of first enrollment	14/05/2026
Target sample size	24
Countries of recruitment
Study type	Interventional
Intervention(s)	Maintenance treatment with efgartigimod (intravenous infusion or subcutaneous injection)

Outcome(s)

Primary Outcome

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 Outcome

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.

Key inclusion & exclusion criteria

Age minimum	>= 18age old
Age maximum	< 80age old
Gender	Both
Include 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 <= 2 - MG Composite score <= 4 and who are receiving a prednisolone-equivalent steroid dose of <= 5 mg/day. 3) Patients who meet either of the following conditions: a) Patients who achieved MM status in a prior study (jRCTs031240366); or b) Patients who are already in MM status in routine clinical practice and are continuing treatment with efgartigimod at the time of enrollment in this study. 4) Patients aged >= 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.
Exclude 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