Contribution of efratigimod to the reduction of oral prednisolone

JRCT ID: jRCTs031240366

Registered date:01/10/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Generalized myasthenia gravis
• Date of first enrollment: 01/10/2024
• Target sample size: 16
• Study type: Interventional
• Intervention(s): Administration as suggested by doctors

Outcome(s)

• Primary Outcome: Reduction in the dose of PSL (mg) from baseline at the study's endpoint

Secondary Outcome

1) Proportion of patients who were able to administer the drug according to the protocol (if not, it is classified according to whether it was due to PSL reduction or side effects.)
2) MG-ADL scale / MG-QOL 15r-J score / MG composite score
3) Responder rate of Efgartigimod (proportion of patients with an improvement of 2 points or more on the MG-ADL scale)
4) Safety of Efgartigimod fixed interval Administration
5) Comparison of clinical course for AchR+,MuSK+,DSNMG subgroup
6) Before and after the test, changes in bone density, HbA1c, and BMI
7) Investigating biomarkers in patients' serum and plasma that can reveal the effectiveness of efgartigimod and the activity of generalized myasthenia gravis

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: < 80age old
• Gender: Both

Include criteria

1) Patients with generalized myasthenia gravis who meet the 2013 diagnostic criteria
2) Patients with generalized myasthenia gravis who are taking more than 6 mg of prednisolone
3) Patients with generalized myasthenia gravis who have an MG-ADL score of over 5
4) Patients whose ages are within 18 to 79
5) Patients who fully understand and agree to participate in the study after receiving a thorough explanation

Exclude criteria

1) Patients who have previously taken efgartigimod
2) Patients who have been treated with other moleculary targeted drugs
3) Patients whose IgG level is below 400 mg/ml during the screening test
4) Patients who are carriers of type B hepatitis virus or have an active HBV/HCV infection
5) Patients with a past history of HBV/HCV infection, excluding those whose nucleic acid levels for HBV/HCV are below the detection limit
6) Patients with severe hepatic dysfunction, indicated by AST (GOT) or ALT (GPT) levels exceeding 100 U/L
7) Patients with severe kidney dysfunction, indicated by serum creatinine level exceeding 2.0 mg/dL
8) Patients with a tuberculosis infection
9) Patients with malignant cancer or a past history of malignant cancer, excluding those who have been recurrence-free for more than 5 years
10) Patients with an active infection
11) Patients who are pregnant or may be pregnant
12) Patients who are breastfeeding
13) Patients who have received a live vaccine within the past 30 days
14) Patients whom the doctors responsible for the study determine are not suited for participation

Related Information

• Primary Sponsor: Saiki Shinji
• Source(s) of Monetary Support: argenx Japan K.K.