Efgartigimod Expanded Access for Generalized Myasthenia Gravis
Efgartigimod Expanded Access for Generalized Myasthenia Gravis
This expanded access protocol applies to patients with gMG who are not enrolled in an ongoing
 clinical trial. The aim of the trial is to provide patients with generalized myasthenia
 gravis (gMG), who are ineligible to participate in a clinical trial, access to efgartigimod
 treatment before regulatory approval.
Inclusion Criteria:

 -  Patient is ≥18 years of age, at the time of signing the informed consent

 -  Patient has a diagnosis of MG (AChR-Ab seropositive or seronegative) with generalized
 muscle weakness

 -  Patient has been vaccinated against COVID-19 or has had a negative COVID-19 test
 result in the 2 weeks before enrollment

 -  Patient has documented IgG >6 g/L within one month of screening

 -  Patient agrees to contraceptive use consistent with local regulations and scientific
 rationale regarding the methods of contraception and:

 1. Male patients: must agree to use an acceptable method of contraception and to not
 donate sperm from the time of providing informed consent until the end of the
 program

 2. Female patients: Women of childbearing potential must use a highly effective or
 acceptable method of contraception and have a negative serum pregnancy test at
 screening

 -  Patient provides signed informed consent, which includes compliance with the
 requirements and restrictions listed in the informed consent form (ICF) and this
 protocol

 -  Patients with a history of hepatitis B, hepatitis C, or HIV must have a documented
 negative test for an active viral infection.

 Exclusion Criteria:

 -  Patient has clinically significant uncontrolled active or chronic bacterial, viral, or
 fungal infection at screening

 -  Patient has a known autoimmune disease that, in the opinion of the treating physician,
 would interfere with an accurate assessment of clinical symptoms of gMG or put the
 patient at undue risk

 -  Patient has a history of malignancy unless it is deemed to be cured by adequate
 treatment with no evidence of recurrence for ≥3 years before the first administration
 of the IMP. Patients with documentation of adequate treatment of the following cancers
 can be included at any time: basal cell or squamous cell skin cancer; carcinoma in
 situ of the cervix; carcinoma in situ of the breast; incidental histological finding
 of prostate cancer (TNM stage T1a or T1b)

 -  Patient has clinical evidence of other significant serious diseases, has recently had
 major surgery, or has any other condition that, in the opinion of the treating
 physician, could put the patient at undue risk

 -  Patient may be excluded based upon review of clinical medical records and screening
 clinical safety laboratory test results

 -  Patient has received a live or a live-attenuated vaccination during the month before
 screening

 -  Patient is pregnant and/or lactating or intends to become pregnant during the program
 or within 90 days after the last dose

 -  Patient is an unsterilized male who is sexually active while participating in the
 program and does not intend to use effective methods of contraception during the
 program through 90 days after the last dose or plans to donate sperm during the
 program or through 90 days after the last dose.

 -  The patient has previously received rituximab, and the last rituximab dose was
 received less than 6 months before the first dose of efgartigimod