A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia

Last updated: May 2, 2025
Sponsor: argenx
Overall Status: Active - Recruiting

Phase

3

Condition

White Cell Disorders

Dysfunctional Uterine Bleeding

Platelet Disorders

Treatment

Efgartigimod IV

Placebo IV

Clinical Study ID

NCT06544499
ARGX-113-2402
2024-515451-38-00
  • Ages > 18
  • All Genders

Study Summary

The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP). After an up to 2 weeks screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV, respectively during the double-blinded treatment period (DBTP). At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first 52-week open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second 52-week OLTP2. After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Is at least 18 years of age and the local legal age of consent for clinical studieswhen signing the informed consent form (ICF).

  • Has documented baseline mean platelet count of <30 x 10^9/L before randomization

  • Has a documented duration of primary immune thrombocytopenia (ITP) of more than 12months on the date of informed consent form (ICF) signature.

  • Has documented prior ITP treatment with at least 1 of the following treatments:corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin,thrombopoietin receptor agonist (TPO-RAs), or rituximab.

  • Has documented insufficient response to a prior ITP treatment (the specific criteriacan be found in the protocol).

  • Has documented prior response defined as 1 platelet count of ≥50 × 109/L to at least 1 of the following ITP treatments in the 3 years before the date of ICF signature:prednisone, dexamethasone, other or nonspecified corticosteroids, IVIg, or anti-Dimmunoglobulin

Exclusion

Exclusion Criteria:

  • Other than the indication under study, known autoimmune disease or any medicalcondition that would interfere with an accurate assessment of clinical symptoms ofITP, confound the results of the study or put the participant at undue risk.

  • Secondary ITP

  • Nonimmune thrombocytopenia

  • Autoimmune hemolytic anemia

  • ITP-associated critical or severe bleeding The complete list of criteria can befound in the protocol.

Study Design

Total Participants: 63
Treatment Group(s): 2
Primary Treatment: Efgartigimod IV
Phase: 3
Study Start date:
October 18, 2024
Estimated Completion Date:
June 30, 2028

Connect with a study center

  • Krankenhaus der Elisabethinen Linz GmbH

    Linz, 4020
    Austria

    Active - Recruiting

  • University Hospital Prof. Dr. Stoyan Kirkovich AD

    Stara Zagora, 6003
    Bulgaria

    Active - Recruiting

  • St James's Hospital - Cancer Clinical Trials Office

    Dublin, D08 NHY1
    Ireland

    Active - Recruiting

  • Azienda ULSS 7 Pedemontana

    Bassano del Grappa, 36061
    Italy

    Active - Recruiting

  • Universita Degli Studi Di Firenze - Azienda Ospedaliero-Universitaria Careggi (AOUC)

    Firenze, 50134
    Italy

    Active - Recruiting

  • AO Maggiore Della Carita

    Novara, 28100
    Italy

    Active - Recruiting

  • U.O.C. Ematologia Ospedale Santa Maria delle Croci

    Ravenna, 48121
    Italy

    Active - Recruiting

  • AO Ordine Mauriziano di Torino

    Torino, 10128
    Italy

    Active - Recruiting

  • Azienda Sanitaria Universitaria Integrata

    Trieste, 34125
    Italy

    Active - Recruiting

  • Pratia Onkologia Katowice

    Katowice, 40-519
    Poland

    Active - Recruiting

  • Complejo Hospitalario Universitario A Coruna

    La Coruna, 15006
    Spain

    Active - Recruiting

  • Hospital Universitario Ramon y Cajal

    Madrid, 28034
    Spain

    Active - Recruiting

  • Hospital General Universitario Morales Meseguer

    Murcia, 30008
    Spain

    Active - Recruiting

  • University Hospital Quironsalud Madrid

    Pozuelo de Alarcon, 28223
    Spain

    Active - Recruiting

  • Hospital Clinico Universitario - University of Valencia

    Valencia, 46010
    Spain

    Active - Recruiting

  • University Hospitals Coventry and Warwickshire NHS Trust

    Coventry, CV2 2DX
    United Kingdom

    Active - Recruiting

  • Glasgow Royal Infirmary - North Glasgow University Hospital Division

    Glasgow, G4 0ET
    United Kingdom

    Active - Recruiting

  • Barts and the London Pathology & Pharmacy Building - Barts Health NHS Trust

    London, E1 2ES
    United Kingdom

    Active - Recruiting

  • Hammersmith Hospital, Imperial College School Of Medicine - Imperial College Healthcare NHS Trust

    London, W12 0HS
    United Kingdom

    Active - Recruiting

  • Royal Cornwall Hospital (RCH)

    Truro, TR1 3LJ
    United Kingdom

    Active - Recruiting

  • University of Southern California Norris Comprehensive Cancer Center

    Los Angeles, California 90033
    United States

    Active - Recruiting

  • Regional Cancer Care Associates, LLC (RCCA)

    Little Silver, New Jersey 07739
    United States

    Active - Recruiting

  • Clinical Research Alliance Inc.

    Westbury, New York 11590
    United States

    Terminated

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