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

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Updated on 10 July 2021


This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in patients with primary ITP.

Condition Primary Immune Thrombocytopenia
Treatment Placebo, efgartigimod
Clinical Study IdentifierNCT04188379
Last Modified on10 July 2021


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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 <3010E9/L from 2 counts: 1 platelet count collected during the screening period and the predose platelet count on the day of randomization (visit 1)
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
Permitted concurrent ITP medications include oral corticosteroids, oral
Patients not receiving concurrent ITP therapy are also eligible for the trial
if they have not received prior ITP therapy for at least 4 weeks prior to
immunosuppressants, dapsone/danazol, fostamatinib, and/or oral TPO-RAs
baseline, and 6 months in case of prior ITP therapy with an anti-CD20 therapy
(eg, rituximab)
\. 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 are considered of
amenorrhea) for at least 1 year with a follicle-stimulating hormone (FSH) of
childbearing potential unless they are postmenopausal (defined by continuous
>40 IU/L or are surgically sterilized (ie, women who had a hysterectomy, a
bilateral salpingectomy, both ovaries surgically removed, or have a documented
permanent female sterilization procedure including tubal ligation). Follicle
\. Women of childbearing potential should use a highly effective or
stimulating hormone can be used to confirm postmenopausal status in
amenorrheic patients on hormonal replacement therapy
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
combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
progestogen-only hormonal contraception associated with inhibition of ovulation
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, diaphragm, or sponge with spermicide. 9. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use an acceptable method of contraception, ie, a condom. Male patients practicing true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant) can be included. Sterilized male patients who have had a vasectomy with documented aspermia post procedure can be included. In addition, male patients are not allowed to donate sperm during this period from signing of ICF, throughout the duration of the trial, and for 90 days after the last administration of 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 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 anti-CD20 therapy (eg, rituximab) within 6 months prior to randomization
Use of romiplostim within 4 weeks prior to randomization
Undergone splenectomy less than 4 weeks prior to randomization
Use of monoclonal antibodies or crystallized fragment (Fc) fusion proteins, other than those previously indicated, within 3 months prior to randomization
At the screening visit, clinically significant laboratory abnormalities as below
Hemoglobin 9 g/dL
Use of any other investigational drug within 3 months or 5 half lives of the drug (whichever is longer) prior to randomization
OR -
International normalized ratio >1.5 or activated partial thromboplastin time >1.5ULN
OR -
Total IgG level <6 g/L
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 (eg, myocardial infarction, stroke, pulmonary embolism, deep venous thrombosis) 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
Patients with known serum-positivity or who test positive for an active viral infection at screening with: Hepatitis B Virus (HBV) (except patients who are anti-HBs Ab positive because of HBV vaccination), Hepatitis C Virus, HIV
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
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
Employees of the investigator or trial center, with direct involvement in the proposed trial or other trials under the direction of that investigator or trial center, as well as family of the employees or the investigator
Patients who received a live/live-attenuated vaccine within 4 weeks before screening. The receipt of any inactivated, sub-unit, polysaccharide, or conjugate vaccine at any time before screening is not considered an exclusion criterion
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