A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP)

Exclusion Criteria

• •ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
• •Use of anticoagulants (eg, vitamin K antagonists, direct oral anticoagulants) within 4 weeks prior to randomization.
• •Use of any transfusions within 4 weeks prior to randomization.
• •Use of Ig (IV, subcutaneous, or intramuscular route) or plasmapheresis (PLEX), 4 weeks prior to randomization.
• •Use of romiplostim within 4 weeks prior to randomization.
• •Undergone splenectomy less than 4 weeks prior to randomization.
• •Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP.
• •Use of any monoclonal antibody within the 6 months before the first dose of the IMP.
• •At the screening visit, clinically significant laboratory abnormalities as below: Hemoglobin ≤9 g/dL - OR - International normalized ratio \>1.5 or activated partial thromboplastin time \>1.5×ULN - OR - Total IgG \<6 g/L.
• •History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Patients with the following cancer 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 finding of prostate cancer (TNM stage T1a or T1b)
• •Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments.
• •History of a major thrombotic or embolic event (eg, myocardial infarction, stroke, deep venous thrombosis, or pulmonary embolism) within 12 months prior to randomization.
• •History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
• •Clinical evidence of other significant serious diseases, have had a recent major surgery, or who have any other condition in the opinion of the investigator, that could confound the results of the trial or put the patient at undue risk.
• •Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV) that is indicative of an acute or chronic infection, Hepatitis C virus (HCV) based on HCV-antibody assay, Human immunodeficiency virus (HIV) based on test results that are associated with an acquired immunodeficiency syndrome (AIDS)-defining condition or a CD4 count \<200 cells/mm3.
• •Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITP (eg, cardiovascular, pulmonary, hematologic, gastrointestinal, endocrine, hepatic, renal, neurological, malignancy, infectious diseases, uncontrolled diabetes) despite appropriate treatments, which could put the patient at undue risk.
• •Known hypersensitivity reaction to efgartigimod or 1 of its excipients.
• •Previously participated in a clinical trial with efgartigimod.
• •Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of the IMP.
• •Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening.
• •Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of ITP or put the patient at undue risk.
• •Current or history of (ie, within 12 months of screening) alcohol, drugs, or medication abuse.