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    EudraCT Number:2019-002100-41
    Sponsor's Protocol Code Number:ARGX-113-1801
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2019-11-25
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - PEI
    A.2EudraCT number2019-002100-41
    A.3Full title of the trial
    A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the efficacy and safety of efgartigimod in adult patients with primary immune thrombocytopenia (an autoimmune disorder that destructs platelets, blood cells that help with clotting, and can lead to easy or excessive bruising and bleeding)
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberARGX-113-1801
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of Sponsorargenx BVBA
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportargenx BVBA
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationargenx BVBA
    B.5.2Functional name of contact pointRegulatory
    B.5.3 Address:
    B.5.3.1Street AddressIndustriepark Zwijnaarde 7
    B.5.3.2Town/ cityZwijnaarde (Ghent)
    B.5.3.3Post code9052
    B.5.4Telephone number+32 9 310 3400
    B.5.5Fax number+32 9 310 3499
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2230
    D.3 Description of the IMP
    D.3.1Product nameEfgartigimod
    D.3.2Product code ARGX-113
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEfgartigimod alpha
    D.3.9.1CAS number 1821402-21-4
    D.3.9.2Current sponsor codeARGX-113
    D.3.9.3Other descriptive nameARGX-113
    D.3.9.4EV Substance CodeSUB198780
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary immune thrombocytopenia
    E.1.1.1Medical condition in easily understood language
    Disorder that can lead to easy or excessive bruising and bleeding due low levels of the cells that help blood clot
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.0
    E.1.2Level LLT
    E.1.2Classification code 10050245
    E.1.2Term Autoimmune thrombocytopenia
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of efgartigimod compared to placebo in achieving a sustained platelet count response in patients with primary chronic 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.
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of efgartigimod compared to placebo in overall platelet count response.
    - To evaluate the safety and tolerability of efgartigimod administered IV weekly or every other week (q2w).
    - To evaluate the incidence and severity of bleeding events while receiving treatment with efgartigimod compared to placebo.
    - To evaluate the use of rescue treatment and changes in concurrent ITP therapy while receiving treatment with efgartigimod compared to placebo.
    - To evaluate the effects of efgartigimod treatment on quality-of-life (QoL) measures and patient-reported outcomes (PRO) compared to placebo.
    - To assess the immunogenicity of efgartigimod.
    - To assess the PK of efgartigimod.
    - To assess the PD effects of efgartigimod.
    E.2.3Trial contains a sub-study No
    E.3Principal 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 ≥18 years.
    3. Confirmed ITP diagnosis, at least 3 months before randomization and according to the ASH Criteria, and no known other 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 <30×10^9/L from 2 counts: 1 platelet count collected during the screening period and the pre-dose platelet count on the day of randomization (visit 1).
    6. 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 immunosuppressants, dapsone/danazol, fostamatinib and/or oral TPO-RAs.
    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 baseline, and 6 months in case of prior ITP therapy with an anti-CD20 therapy (eg, rituximab).
    7. Women of childbearing potential must have a negative serum pregnancy test at the screening visit and a negative urine pregnancy test at baseline before study medication (infusion) can be administered. Women are considered of childbearing potential unless they are post-menopausal (defined by continuous amenorrhea) for at least 1 year with an FSH of >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-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:
    - 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, 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.
    E.4Principal exclusion criteria
    1. ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia
    associated with myeloid dysplasia.
    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, subcutaneous or intramuscular route) or plasmapheresis (PLEX), 4 weeks prior to randomization.
    5. Use of anti-CD20 therapy (eg, 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 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 ≤9 g/dL.
    - International normalized ratio >1.5 or activated partial thromboplastin time >1.5×ULN.
    - Total IgG level <6 g/L.
    11. 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 nonmelanoma 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 eg, brain, heart, lung, liver, bladder, or gastrointestinal tract)
    within 4 weeks of randomization.
    16. 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.cd.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 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 (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.
    18. Patients with known medical history of hypersensitivity to any of the ingredients of the IMP.
    19. Patients who previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the IMP.
    20. Pregnant or lactating females.
    21. 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.
    22. 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.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients with chronic ITP with a sustained platelet count response defined as achieving 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.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Between week 19 and 24 of the trial.
    E.5.2Secondary end point(s)
    Key Secondary Efficacy Endpoints Subject to Alpha Control:
    1. Extent of disease control defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10^9/L in the chronic ITP population.
    2. Proportion of patients in the overall population (chronic and persistent ITP) with a sustained platelet count response defined as achieving 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.
    3. Incidence and severity of the WHO-classified bleeding events in the overall population.
    4. Proportion of patients in the overall population achieving platelet counts of at least 50×10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Over the 24-week treatment
    2. Between week 19 and 24
    3. At each visit
    4. Between week 17 and 24
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Tolerability, immunogenicity of efgartigimod
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA75
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Russian Federation
    United Kingdom
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    End-of-Trial is defined as last patient last visit in the trial.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days7
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 140
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 16
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 140
    F.4.2.2In the whole clinical trial 156
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Patients completing the 24 week randomized trial period will perform the End-of-Treatment visit and can enter the open-label extension trial (ARGX 113 1803) to receive efgartigimod 10 mg/kg IV.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-02-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-04-14
    P. End of Trial
    P.End of Trial StatusOngoing
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