A Phase 3 study of Efgartigimod PH20 SC in patient with Generalized Myasthenia Gravis

JRCT ID: jRCT2021210016

Registered date:01/07/2021

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Generalized Myasthenia Gravis (gMG)
• Date of first enrollment: 12/05/2021
• Target sample size: 10
• Countries of recruitment: Belgium,Japan,Czech Republic,Japan,Germany,Japan,Hungary,Japan,Italy,Japan,Netherlands,Japan,Poland,Japan,Spain,Japan,Bosnia and Herzegovina,Japan,Georgia,Japan,Serbia,Japan,Russia,Japan,the UK,Japan,the US,Japan
• Study type: Interventional
• Intervention(s): Subcutaneous administration of Efgartigimod PH20 SC or intravenous administration of ARGX-113 once a week

Outcome(s)

• Primary Outcome: Percent reduction from baseline in total IgG levels at day 29, ie, 7 days after the fourth IV or SC administration

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
2. At least 20 years of age at the time of signing the ICF.
3. Diagnosed with gMG with confirmed documentation and supported by at least 1 of the following:
a. History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or
repetitive nerve stimulation
b. History of positive edrophonium chloride test
c. Demonstrated improvement in MG signs upon treatment with oral acetylcholinesterase (AChE) inhibitors
as assessed by the treating physician
4. Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II,
III, IVa, or IVb

Exclude criteria

1. Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last d
ose of IMP.
2. Has any of the following medical conditions:
a. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
b. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an ac
curate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk
c. History of malignancy unless deemed cured by adequate treatment with no evidence of reoccurrence f
or >=3 years before the first administration of the IMP. Participants with the following cancers can be
included at any time:
- adequately treated basal cell or squamous cell skin cancer
- carcinoma in situ of the cervix
- carcinoma in situ of the breast
- incidental histological findings of prostate cancer (TNM Classification of Malignant Tumors stage T1a
or T1b).
d. Clinical evidence of other significant serious diseases, or the participant has had a recent major surger
y, or who have any other condition that, in the opinion of the investigator, could confound the results of
the study or put the participant at undue risk.

Related Information

• Primary Sponsor: Matsuura Yuji