a study to assess the efficacy and safety of efgartigimod in adult patients with primary immune thrombocytopenia (itp).

Conditions: Primary Immune Thrombocytopenia

Summary: this is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of argx-113 in participants with primary itp.


Eligibility criteria:
inclusion criteria: * ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits). * male or female patient aged ≥18 years. * confirmed itp diagnosis, at least 3 months before randomization and according to the american society of hematology criteria, and no known other etiology for thrombocytopenia. * diagnosis supported by a response to a prior itp therapy (other than thrombopoietin receptor agonists \[tpo-ras\]), in the opinion of the investigator. * mean platelet count of \<30×10e9/l from 2 counts: 1 platelet count collected during the screening period and the predose platelet count on the day of randomization. * at the start of the trial, the patient is either on concurrent itp treatment(s) and has received at least 1 prior therapy for itp in the past, or the patient is not on treatment for itp but has received at least 2 prior treatments for itp. patients receiving permitted concurrent itp treatment(s) at baseline, must have been stable in dose and frequency for at least 4 weeks prior to randomization. * women of childbearing potential must have a negative serum pregnancy test at the screening visit and a negative urine pregnancy test at baseline before trial medication (infusion) can be administered. * women of childbearing potential should use a highly effective or acceptable method of contraception during the trial and for 90 days after the last administration of the imp. 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 certain medications before the start of the studies (more details in the protocol) * patients who have a history of malignancy, including malignant thymoma, or myeloproliferative or lymphoproliferative disorders, unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before screening. patients with completely excised non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted at any time. * uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmhg (systolic) and/or 100 mmhg (diastolic) despite appropriate treatments. * history of any major thrombotic or embolic event within 12 months prior to randomization. * history of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia. * history of a recent or planned major surgery (that involves major organs eg, brain, heart, lung, liver, bladder, or gastrointestinal tract) within 4 weeks of randomization. * positive serum test at screening for an active viral infection with any of the following conditions: hepatitis b virus (hbv), hepatitis c virus (hcv), human immunodeficiency virus (hiv) * clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than itpdespite appropriate treatments which could put the patient at undue risk. * patients with known medical history of hypersensitivity to any of the ingredients of the imp. * patients who previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the imp. * pregnant or lactating females. more details in the protocol

Keywords: immune thrombocytopenia sustained platelet count response bleeding events