Primary immune thrombocytopenia
Conditions
Interventions
Patients will receive 10 mg/kg of efgartigimod or placebo, weekly from visits 1 to 4 and then from visits 5 to 16 either weekly or biweekly, adjusted according to their platelet count. From visits 17
Sponsors
Jaume Ayguasanosa
Eligibility
Inclusion criteria
Inclusion criteria: 1. 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). 2. Male or female patient aged >=20 years. 3. 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. 4. Diagnosis supported by a response to a prior ITP therapy (other than thrombopoietin receptor agonists [TPO RAs]), in the opinion of the investigator. 5. Mean platelet count of 40 IU/L or are surgically sterilized (i.e. women who had a hysterectomy, a bilateral salpingectomy, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle-stimulating hormone can be used to confirm post-menopausal status in amenorrheic patients not on hormonal replacement therapy. 8. 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. They must be on a stable regimen, for at least 1 month: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable), intrauterine device (IUD), intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner (provided that the partner is the sole sexual partner of the trial participant and documented aspermia post procedure), continuous abstinence from heterosexual sexual contact. Sexual abstinence is only allowable if it is the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, symptothermal, post ovulation methods) is not acceptable. Male or female condom with or without spermicide. Cap, diapfragm, or sponge with spermicide. 9. Non-sterilized male patients who are sexually active with a female partner of childbea
Exclusion criteria
Exclusion criteria: 1. ITP/thrombocytopenia associated with another condition, e.g. lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia. 2. Use of anticoagulants (e.g. 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, subcutaneous, or intramuscular route), or plasmapheresis (PLEX), 4 weeks prior to randomization. 5. Use of anti-CD20 therapy (e.g. rituximab) within 6 months prior to randomization. 6. Use of romiplostim within 4 weeks prior to randomization. 7. Undergone splenectomy less than 4 weeks prior to randomization. 8. Use of any other investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to randomization. 9. Use of monoclonal antibodies or crystallized fragment (Fc) fusion proteins, other than those previously indicated, within 3 months prior to randomization. 10. At the screening visit, clinically significant laboratory abnormalities as below: Hemoglobin 1.5 or activated partial thromboplastin time >1.5*ULN. - OR - Total IgG level =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. 12. Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments. 13. History of any major thrombotic or embolic event (eg, myocardial infarction, stroke, pulmonary embolism, deep venous thrombosis) within 12 months prior to randomization. 14. History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia. 15. History of a recent or planned major surgery (that involves major organs e.g. brain, heart, lung, liver, bladder, or gastrointestinal tract) within 4 weeks of randomization. 16. Patients with known serum-positivity or who test Positive serum test at screening for an active viral infection at screening 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)( (except patients who are anti-HBs Ab positive because of HBV vaccination), Hepatitis C Virus, HIV. 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 immunodeficiency syndrome (AIDS)-defining condition or a CD4 count <=200 cells/mm^3 17. Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITP (e.g. cardiovascular, pulmonary, hematologic, gastrointestinal, endocrine, hepatic, renal, neurological, malignancy, infectious diseases, uncontrolled diabetes) despite appropriate treatments which could put the patient at undue risk. 18. Patients with known medical history of hypersensitivity to any of the ingredients of the IMP. 19. Patients who previous
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the efficacy of efgartigimod compared to placebo in achieving a sustained platelet count response in patients with chronic primary immune thrombocytopenia (ITP), with a sustained platelet count response defined as platelet counts of at least 50*10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial. | — |
Countries
Austria, Belgium, Bulgaria, France, Georgia, Germany, Hungary, Italy, Japan, Poland, Russia, Spain, The Czech Republic, The Netherlands, The United Kingdom, The United States, Turkey, Ukraine
Contacts
Public ContactRegulatory Affairs ICON Japan
ICON Japan
Outcome results
None listed