IRB Study Number 25-490
Status Recruiting
Phase Phase 4
Location Cleveland Clinic Main Campus
Institute Neurological Institute
Description
Efgartigimod is being tested in this study, may help improve Myasthenia Gravis (MG), and has been approved as a treatment for adult patients with MG by the United States Food and Drug Administration.
The study will last about 58 weeks and there are at least 33 visits during the study, and at least 20 of these will be at the study site. In this study, certain visits will occur over the phone. Each study site visit will last about 2 hours, and each phone visit will last less than 1 hour.
Inclusion Criteria
Participants can be included in the study only if all the following criteria apply:
- Is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF
- Is capable of providing signed informed consent, and can comply with protocol requirements
- If a Woman of Childbearing Potential (WOCBP), agrees to use contraceptive measures consistent with local regulations and study requirements.
- If a WOCBP , has a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before receiving Investigational Product (IMP).
- Has been diagnosed with gMG of MGFA class II, III, or IV
- Is seropositive for acetylcholine receptor (AChR) antibodies, confirmed at screening via: Examination of medical records documenting previous seropositive result; or AChR-Ab serological assessment performed at screening
- Has 1 of the following associated with their gMG diagnosis: Abnormal Repetitive nerve stimulation (RNS) test result or abnormal Single-fiber electromyography (SF-EMG) test result consistent with gMG or clinically meaningful response to Acetylcholinesterase inhibitors (AChEI) therapy
- Is treatment-naive for gMG or has been administered AChEI for the treatment of gMG
- Had onset of generalized MG signs and/or symptoms within 12 months before screening; candidates who also had onset of ocular MG signs and/or symptoms within 24 months before screening may be enrolled in the study.
- Has an MG Activities of Daily Living (MG-ADL) score ≥5 at screening and baseline
Exclusion Criteria
Participants will be excluded from the study if any of the following criteria apply:
- gMG diagnosis of Myasthenia Gravis Foundation of America (MGFA) class I or V
- Underwent a thymectomy prior to screening (exception: study candidates who underwent a thymectomy for treatment of nonmalignant thymoma before they were diagnosed with gMG may be enrolled in the study)
- Prior or current use of any of the following immunosuppressive therapies for the treatment of gMG: Any systemic corticosteroid therapy (CST) within 12 months before screening (inhaled and topical CST are permitted at any time before and throughout the study) or any Nonsteroidal Immunosuppressant Therapy (NSIST), (including, but not limited to, azathioprine, cyclophosphamide, cyclosporin, methotrexate, mycophenolate mofetil) before screening
- Other than the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of gMG or puts the participant at undue risk
- History of malignancy unless considered cured by adequate treatment: with no evidence of recurrence for ≥3 years before first IMP administration of the following cancers: 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 stage T1a or T1b)
- Serious or severe active infection that is not sufficiently resolved before baseline in the investigator’s opinion
- Positive serum test result at screening for active infection with any of the following: hepatitis B virus (HBV) indicative of an acute or chronic infection unless associated with a negative HBV DNA test result, hepatitis C virus (HCV) based on HCV antibody assay unless a negative RNA test result is available, HIV based on test results of a CD4 count <200 cells/mm3, Having an AIDS-defining condition, HIV based on test results of a CD4 count ³200 cells/mm3 but not adequately treated with antiviral therapy
- Disease or any other medical condition that, in the investigator’s opinion, would confound the study results or put the participants at undue risk.
- Recent major surgery (within 3 months of screening) or intention to have major surgery during the study
- Different IMP received in another clinical study <12 weeks or <5 half-lives (whichever is longer) before screening
- Current participation in another interventional clinical study
- Known hypersensitivity to IMP or 1 of its excipients
- History (within 12 months before screening) of or current alcohol, drug, or medication abuse as assessed by the investigator
- Pregnant or lactating state or intention to become pregnant during the study
- Previous participation in an efgartigimod clinical study and at least 1 dose of IMP received, or previous exposure to at least 1 dose of efgartigimod in medical practice
- Total IgG concentration <4 g/L at screening
- Monoclonal antibody that is not an anti-CD20 or Fc-fusion protein received <4 weeks before screening
- Complement inhibitors (including but not limited to eculizumab, ravulizimab, and zilucoplan) administered <24 months before screening
- Anti-CD20 or anti-CD19 antibody received <6 months before screening
- Intravenous immunoglobulin (IVIg), subcutaneous immunoglobulin (SCIg), or plasma exchange (PLEX) received <4 weeks before screening
- Live or live-attenuated vaccine received <4 weeks before screening
- Severe renal impairment with an estimated glomerular filtration rate of <30 mL/min/1.73 m2 at screening
