Primary Immune Thrombocytopenia
Conditions
Interventions
Patients will receive ARGX-113 PH20 SC (1000 mg of Efgartigimod Alfa) or placebo PH20 SC, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or biweekly, adjusted according to their
Sponsors
Gandini Domenica
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the trial only if all of the following criteria apply: 1. Ability to understand the requirements of the trial and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits) 2. Male or female, aged >=18 years at the time the informed consent form (ICF) is signed 3. Confirmed diagnosis of primary ITP made at least 3 months before randomization and based on the American Society of Hematology Criteria, and no known etiology for thrombocytopenia 4. Diagnosis supported by a response to a prior ITP therapy (other than TPO-RAs), in the opinion of the investigator 5. Mean platelet count of 10 years, and currently lives in Japan.
Exclusion criteria
Exclusion criteria: Participants are excluded from the trial if any of the following criteria apply: 1. Secondary ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, thrombocytopenia associated with myeloid dysplasia, or hematopoietic stem cell transplant 2. Use of anticoagulants (eg, vitamin K antagonists, direct oral anticoagulants) within 4 weeks prior to randomization 3. Use of any transfusions within 4 weeks prior to randomization 4. Use of Ig (IV, SC, or intramuscular route) or plasmapheresis (PLEX) within 4 weeks prior to randomization 5. Use of romiplostim within 4 weeks prior to randomization 6. Undergone splenectomy less than 4 weeks prior to randomization 7. Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of the IMP 8. Use of any monoclonal antibody or Fc fusion proteins, other than those previously indicated, within 6 months before the first dose of the IMP (eg, anti-CD20) 9. At the screening visit, clinically significant laboratory abnormalities as follows: - Hemoglobin 1.5 or activated partial thromboplastin time >1.5*upper limit of normal OR - total IgG level 3 years before the first administration of IMP. Participants with the following cancer can be included at any time: a. Adequately treated basal cell or squamous cell skin cancer b. Carcinoma in situ of the cervix c. Carcinoma in situ of the breast or d. Incidental histological finding of prostate cancer (TNM stage T1a or T1b) 11. Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments 12. History of any major thrombotic or embolic event (eg, myocardial infarction, stroke, deep venous thrombosis, or pulmonary embolism) within 5 years prior to randomization 13. History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia 14. Evidence of an active clinically significant bleeding of an organ or internal mucosal bleeding, other than expected in ITP, that warrants emergent treatment or therapeutic procedure based on the judgment of Investigator (eg, intracranial hemorrhage, pulmonary hemorrhage, bleeding with ongoing need for packed red blood cell transfusion) 15. Estimated high risk of a clinically significant bleeding of an organ or internal mucosal bleeding, other than expected in ITP, that warrants emergent treatment or therapeutic procedure according to the judgment of Investigator 16. 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 participant at undue risk 17. Positive serum test at screening for an active viral infection with any of the following conditions: a. Hepatitis B virus (HBV) that is indicative of an acute or chronic infection, unless associated with a negative HBV DNA test (https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf) b. Hepatitis C virus (HCV) based on HCV-antibody assay (unless associated with a negative HCV RNA test) c. Human immunodeficiency virus (HIV) based on test results that are associated with an acquired immu
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of patients with chronic ITP with a sustained platelet count response defined as achieving platelet counts of >=50*10^9/L for at least 4 of the 6 visits between week 19 and week 24 of the trial. | — |
Countries
Argentina, Australia, Bulgaria, Chile, China, Colombia, Denmark, France, Georgia, Germany, Greece, Ireland, Israel, Italy, Japan, Jordan, Korea, Mexico, New Zealand, Norway, Peru, Poland, Portugal, Republic of Latvia, Romania, Russian Federation, Serbia, South Africa, Spain, Taiwan, Thailand
Contacts
Public ContactClinical trial contact
ICON Clinical Research GK
Outcome results
None listed