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

NCT ID: NCT06544499

Last Updated: 2026-02-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

69 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-18

Study Completion Date

2028-06-30

Brief Summary

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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.

Detailed Description

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Conditions

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Primary Immune Thrombocytopenia (ITP)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Efgartigimod IV

Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s)

Group Type EXPERIMENTAL

Efgartigimod IV

Intervention Type BIOLOGICAL

Intravenous infusion of efgartigimod

Placebo IV

Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s)

Group Type EXPERIMENTAL

Efgartigimod IV

Intervention Type BIOLOGICAL

Intravenous infusion of efgartigimod

Placebo IV

Intervention Type OTHER

Intravenous infusion of placebo

Interventions

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Efgartigimod IV

Intravenous infusion of efgartigimod

Intervention Type BIOLOGICAL

Placebo IV

Intravenous infusion of placebo

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Is at least 18 years of age and the local legal age of consent for clinical studies when 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 12 months 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 criteria can 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-D immunoglobulin

Exclusion Criteria

* Other than the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of ITP, 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 be found in the protocol.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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argenx

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Mayo Clinic Hospital Scottsdale

Phoenix, Arizona, United States

Site Status RECRUITING

University of Southern California Norris Comprehensive Cancer Center

Los Angeles, California, United States

Site Status RECRUITING

Sharp Memorial Hospital

Oceanside, California, United States

Site Status RECRUITING

University of Colorado Hospital

Aurora, Colorado, United States

Site Status RECRUITING

Yale University School of Medicine

New Haven, Connecticut, United States

Site Status RECRUITING

Georgetown University Hospital

Washington D.C., District of Columbia, United States

Site Status RECRUITING

The University of Chicago Medicine

Chicago, Illinois, United States

Site Status RECRUITING

The University of Iowa Hospitals & Clinics

Iowa City, Iowa, United States

Site Status RECRUITING

Tulane University

New Orleans, Louisiana, United States

Site Status RECRUITING

Henry Ford Health

Detroit, Michigan, United States

Site Status RECRUITING

University of Minnesota

Minneapolis, Minnesota, United States

Site Status RECRUITING

Regional Cancer Care Associates, LLC (RCCA)

Little Silver, New Jersey, United States

Site Status RECRUITING

Clinical Research Alliance Inc.

Westbury, New York, United States

Site Status TERMINATED

Duke University Medical Center

Durham, North Carolina, United States

Site Status RECRUITING

University Hospitals Cleveland Medical Center

Cleveland, Ohio, United States

Site Status RECRUITING

Ohio State University Hospital East

Columbus, Ohio, United States

Site Status RECRUITING

INTEGRIS Cancer Institute of Oklahoma

Oklahoma City, Oklahoma, United States

Site Status RECRUITING

Allgemeines Krankenhaus der Stadt Linz

Linz, , Austria

Site Status RECRUITING

Ordensklinikum Linz GmbH Elisabethinen

Linz, , Austria

Site Status RECRUITING

Klinikum Kreuzschwestern Wels

Wels, , Austria

Site Status RECRUITING

Acibadem City clinic Tokuda hospital

Sofia, , Bulgaria

Site Status RECRUITING

UMHAT Aleksandrovska

Sofia, , Bulgaria

Site Status RECRUITING

University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski

Sofia, , Bulgaria

Site Status RECRUITING

National Specialized Hospital for Active Treatment of Hematology Diseases

Sofia, , Bulgaria

Site Status RECRUITING

UMHAT SofiaMed

Sofia, , Bulgaria

Site Status RECRUITING

University Hospital Prof. Dr. Stoyan Kirkovich AD

Stara Zagora, , Bulgaria

Site Status RECRUITING

Beijing YouYi Hospital-Beijing Friendship Hospital Capital Medical University

Beijing, , China

Site Status RECRUITING

Peking University Third Hospital

Beijing, , China

Site Status RECRUITING

The First Affiliated Hospital Of Bengbu Medical College

Bengbu, , China

Site Status RECRUITING

The Affiliated Hospital of Chengde Medical University

Chengde, , China

Site Status RECRUITING

The Affiliated Hospital of Inner Mongolia Medical University

Hohhot, , China

Site Status RECRUITING

The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University

Huai'an, , China

Site Status RECRUITING

Huizhou Central People's Hospital

Huizhou, , China

Site Status RECRUITING

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College

Tianjin, , China

Site Status RECRUITING

Union Hospital Tongji Medical College of Huazhong University of Science and Technology (HUST)

Wuhan, , China

Site Status RECRUITING

Wuxi People's Hospital (Wuxi No.1 People's Hospital)

Wuxi, , China

Site Status RECRUITING

Affiliated Hospital of Jiangsu University

Zhenjiang, , China

Site Status RECRUITING

University Hospital Center Zagreb

Zagreb, , Croatia

Site Status RECRUITING

Fakultni Nemocnice Ostrava

Ostrava, , Czechia

Site Status RECRUITING

Fakultni nemocnice Kralovske Vinohrady

Prague, , Czechia

Site Status RECRUITING

CHU d'Amiens-Picardie - Hopital SUD

Amiens, , France

Site Status RECRUITING

Centre Hospitalier Universitaire (CHU) - Hopital Henri Mondor

Créteil, , France

Site Status RECRUITING

Centre Hospitalier Universitaire Dijon Bourgogne Medecine Interne et Immunologie Clinique

Dijon, , France

Site Status RECRUITING

University Hospital Carl Gustav Carus

Dresden, , Germany

Site Status RECRUITING

Universitaetsklinikum Essen (AoR)

Essen, , Germany

Site Status RECRUITING

Semmelweis University-Internal Medicine and Haematology

Budapest, , Hungary

Site Status RECRUITING

Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz

Győr, , Hungary

Site Status RECRUITING

Mater Misericordiae University Hospital

Dublin, , Ireland

Site Status RECRUITING

St James's Hospital - Cancer Clinical Trials Office

Dublin, , Ireland

Site Status RECRUITING

Azienda ULSS 7 Pedemontana

Bassano del Grappa, , Italy

Site Status RECRUITING

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

Florence, , Italy

Site Status RECRUITING

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Milan, , Italy

Site Status RECRUITING

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Milan, , Italy

Site Status RECRUITING

ASST Grande Ospedale Metropolitano Niguarda

Milan, , Italy

Site Status RECRUITING

AO Maggiore Della Carita

Novara, , Italy

Site Status RECRUITING

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

Ravenna, , Italy

Site Status RECRUITING

Arcispedale Santa Maria Nuova (ASMN) Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS)

Reggio Emilia, , Italy

Site Status RECRUITING

A.O.U. Citta della Salute e della Scienza di Torino

Torino, , Italy

Site Status RECRUITING

AO Ordine Mauriziano di Torino

Torino, , Italy

Site Status RECRUITING

Azienda Sanitaria Universitaria Integrata

Trieste, , Italy

Site Status RECRUITING

ASST Sette Laghi Ospedale di circolo e Fondazione Macchi

Varese, , Italy

Site Status RECRUITING

Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy

Bydgoszcz, , Poland

Site Status RECRUITING

Pratia Onkologia Katowice

Katowice, , Poland

Site Status RECRUITING

Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi

Lodz, , Poland

Site Status RECRUITING

NZOZ Neuromed M. i M. Nastaj Sp.P.

Lublin, , Poland

Site Status RECRUITING

Uniwersytecki Szpital Kliniczny Nr 4 w Lublinie Centrum Innowacyjnych Terapii

Lublin, , Poland

Site Status RECRUITING

Szpital Specjalistyczny im. Jedrzeja Sniadeckiego w Nowym Saczu

Nowy Sącz, , Poland

Site Status RECRUITING

Wojewodzki Szpital Zespolony im. Ludwika Rydygiera w Toruniu

Torun, , Poland

Site Status RECRUITING

Unidade Local de Saude de Braga

Braga, , Portugal

Site Status RECRUITING

Unidade Local de Saude Gaia e Espinho, E.P.E.

Vila Nova de Gaia, , Portugal

Site Status RECRUITING

Spitalul Clinic Coltea, Clinica de Hematologie

Bucharest, , Romania

Site Status RECRUITING

Spitalul Clinic Colentina

Bucharest, , Romania

Site Status RECRUITING

Spitalul Clinic Judetean de Urgenta Sibiu (SCJS) (Clinical County Hospital Sibiu)

Sibiu, , Romania

Site Status RECRUITING

Spitalul Clinic Judetean de Urgenta Targu-Mures

Târgu Mureş, , Romania

Site Status RECRUITING

Clinical Center of Serbia

Belgrade, , Serbia

Site Status RECRUITING

University Clinical Centre of Kragujevac

Kragujevac, , Serbia

Site Status RECRUITING

Clinical Center of Nis

Niš, , Serbia

Site Status RECRUITING

University Clinical Hospital Center Zemun

Zemun, , Serbia

Site Status RECRUITING

General Hospital Djordje Joanovic

Zrenjanin, , Serbia

Site Status RECRUITING

Complejo Hospitalario Universitario A Coruna

A Coruña, , Spain

Site Status RECRUITING

Instituto Catalan de Oncologia - Hospital Duran i Reynals

L'Hospitalet de Llobregat, , Spain

Site Status RECRUITING

Hospital Universitario Ramon y Cajal

Madrid, , Spain

Site Status RECRUITING

Hospital General Universitario Morales Meseguer

Murcia, , Spain

Site Status RECRUITING

University Hospital Quironsalud Madrid

Pozuelo de Alarcón, , Spain

Site Status RECRUITING

Hospital Clinico Universitario - University of Valencia

Valencia, , Spain

Site Status RECRUITING

Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust

Birmingham, , United Kingdom

Site Status RECRUITING

Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation

Bradford, , United Kingdom

Site Status RECRUITING

University Hospitals Coventry and Warwickshire NHS Trust

Coventry, , United Kingdom

Site Status RECRUITING

Glasgow Royal Infirmary - North Glasgow University Hospital Division

Glasgow, , United Kingdom

Site Status RECRUITING

University Hospitals of Leicester NHS Trust-Leicester Royal Infirmary

Leicester, , United Kingdom

Site Status RECRUITING

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

London, , United Kingdom

Site Status RECRUITING

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

London, , United Kingdom

Site Status RECRUITING

Royal Cornwall Hospital (RCH)

Truro, , United Kingdom

Site Status RECRUITING

Countries

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United States Austria Bulgaria China Croatia Czechia France Germany Hungary Ireland Italy Poland Portugal Romania Serbia Spain United Kingdom

Central Contacts

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Sabine Coppieters, MD

Role: CONTACT

857-350-4834

Facility Contacts

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Surbhi Shah, MD

Role: primary

857-350-4834

Caroline I Piatek, MD

Role: primary

857-350-4834

Catherine Quinn, MD

Role: primary

857-350-4834

Gemlyn George, MD

Role: primary

857-350-4834

Yevgeniya Foster, MD

Role: primary

857-350-4834

Catherine Broome, MD

Role: primary

857-350-4834

Michael Drazer, MD

Role: primary

857-350-4834

Steven Lentz, MD

Role: primary

857-350-4834

Maissaa Janbain, MD

Role: primary

857-350-4834

Vrushali Dabak, MD

Role: primary

857-350-4834

Marshall Mazepa, MD

Role: primary

857-350-4834

Iuliana Shapira, MD

Role: primary

857-350-4834

Ara Metjian, MD

Role: primary

857-350-4834

Koen van Besien, MD

Role: primary

857-350-4834

Marina Beltrami Moreira, MD

Role: primary

857-350-4834

Bashar Alasad, MD

Role: primary

857-350-4834

Clemens Schmitt, MD

Role: primary

857-350-4834

Robert Milanov, MD

Role: primary

857-350-4834

Sonja Heibl, MD

Role: primary

857-350-4834

Ismail Amine, MD

Role: primary

857-350-4834

Evgueniy Hadjiev, MD

Role: primary

857-350-4834

Atanas Radinoff, MD

Role: primary

857-350-4834

Tanya Yankova, MD

Role: primary

857-350-4834

Tzvetan Alaikov, MD

Role: primary

857-350-4834

Maria Todorova, MD

Role: primary

857-350-4834

Zhao Wang, MD

Role: primary

857-350-4834

Hongmei Jing, MD

Role: primary

857-350-4834

Feng Zhang, MD

Role: primary

857-350-4834

Zhihua Zhang, MD

Role: primary

857-350-4834

Da Gao, MD

Role: primary

857-350-4834

Chunling Wang, MD

Role: primary

857-350-4834

Guowei Li, MD

Role: primary

857-350-4834

Lei Zhang, MD

Role: primary

857-350-4834

Heng Mei, MD

Role: primary

857-350-4834

Yun Zhuang, MD

Role: primary

857-350-4834

Xiaoming Fei, MD

Role: primary

857-350-4834

Drazen Pulanic, MD

Role: primary

857-350-4834

Ivo Demel, MD

Role: primary

857-350-4834

Olga Cerna, MD

Role: primary

857-350-4834

Clement Gourguechon, MD

Role: primary

857-350-4834

Marc Michel, MD

Role: primary

857-350-4834

Bernard Bonnotte, MD

Role: primary

857-350-4834

Karolin Trautmann, MD

Role: primary

857-350-4834

Role: backup

Alexander Roeth, MD

Role: primary

857-350-4834

Zsolt Nagy, MD

Role: primary

857-350-4834

Zsolt Lazar, MD

Role: primary

857-350-4834

Anne Fortune, MD

Role: primary

857-350-4834

Role: backup

Catherine M Flynn, MD

Role: primary

857-350-4834

Eros Di Bona, MD

Role: primary

857-350-4834

Valentina Carrai, MD

Role: primary

857-350-4834

Andrea Artoni, MD

Role: primary

857-350-4834

Role: backup

Bruno Fattizzo, MD

Role: primary

857-350-4834

Role: backup

Monica Carpenedo, MD

Role: primary

857-350-4834

Andrea Patriarca, MD

Role: primary

857-350-4834

Claudia Cellini, MD

Role: primary

857-350-4834

Barbara Gamberi, MD

Role: primary

857-350-4834

Role: backup

Alessandra Borchiellini, MD

Role: primary

857-350-4834

Piera Sivera, MD

Role: primary

857-350-4834

Francesco Zaja, MD

Role: primary

857-350-4834

Domenica Caramazza, MD

Role: primary

857-350-4834

Role: backup

Marta Sobas, MD

Role: primary

857-350-4834

Sebastian Grosicki, MD

Role: primary

857-350-4834

Jacek Trelinski, MD

Role: primary

857-350-4834

Role: backup

Tomasz Gromek, MD

Role: primary

857-350-4834

Role: backup

Marta Morawska, MD

Role: primary

857-350-4834

Szymon Fornagiel, MD

Role: primary

857-350-4834

Role: backup

Marcin Rymko, MD

Role: primary

857-350-4834

Carlos Alberto Pereira Capela, MD

Role: primary

857-350-4834

Teresa Melo, MD

Role: primary

857-350-4834

Gabriela Borsaru, MD

Role: primary

857-350-4834

Role: backup

Georgeta Daniela Georgescu, MD

Role: primary

857-350-4834

Role: backup

Alina Catana, MD

Role: primary

857-350-4834

Role: backup

Ioan Macarie, MD

Role: primary

857-350-4834

Role: backup

Mirjana Mitrovic, MD

Role: primary

857-350-4834

Danijela Jovanovic, MD

Role: primary

857-350-4834

Ivan Tijanic, MD

Role: primary

857-350-4834

Zorica Cvetkovic, MD

Role: primary

857-350-4834

Nebojsa Rajic, MD

Role: primary

857-350-4834

Role: backup

Maria del Carmen Gomez del Castillo Solano, MD

Role: primary

857-350-4834

Silvana Ana Novelli Canales, MD

Role: primary

857-350-4834

Role: backup

Francisco Javier Lopez Jimenez, MD

Role: primary

857-350-4834

Maria Luisa Lozano Almela, MD

Role: primary

857-350-4834

Maria Aranzazu Alonso Alonso, MD

Role: primary

857-350-4834

Maria Luisa Calabuig Munoz, MD

Role: primary

857-350-4834

Charles Percy, MD

Role: primary

857-350-4834

Samuel Ackroyd, MD

Role: primary

857-350-4834

Role: backup

Benjamin Bailiff, MD

Role: primary

857-350-4834

Catherine Bagot, MD

Role: primary

857-350-4834

Mamta Garg, MD

Role: primary

857-350-4834

Frederick Chen, MD

Role: primary

857-350-4834

Nichola Cooper, MD

Role: primary

857-350-4834

Adam Forbes, MD

Role: primary

857-350-4834

Other Identifiers

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2024-515451-38-00

Identifier Type: CTIS

Identifier Source: secondary_id

ARGX-113-2402

Identifier Type: -

Identifier Source: org_study_id

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