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NCT06544499PHASE3Recruiting

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

argenx

Start Date

10/18/2024

Completion Date

6/1/2028

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. More information can be found here: https://clinicaltrials.argenx.com/advancenext

Eligibility Criteria

Age Range: 18 years to No maximum

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.

Interventions

BIOLOGICAL

Efgartigimod IV

OTHER

Placebo IV

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Conditions

Primary Immune Thrombocytopenia (ITP)

Locations

Mayo Clinic Hospital Scottsdale

Phoenix, Arizona 85054

United States

University of Southern California Norris Comprehensive Cancer Center

Los Angeles, California 90033

United States

Sharp Memorial Hospital

Oceanside, California 92056

United States

University of Colorado Hospital

Aurora, Colorado 80045

United States

Yale University School of Medicine

New Haven, Connecticut 06510

United States

Georgetown University Hospital

Washington D.C., District of Columbia 20007

United States

The University of Chicago Medicine

Chicago, Illinois 60637

United States

The University of Iowa Hospitals & Clinics

Iowa City, Iowa 52242

United States

Tulane University

New Orleans, Louisiana 70112

United States

Henry Ford Health

Detroit, Michigan 48202

United States

University of Minnesota

Minneapolis, Minnesota 55422

United States

Regional Cancer Care Associates, LLC (RCCA)

Little Silver, New Jersey 07739

United States

Clinical Research Alliance Inc.

Westbury, New York 11590

United States

Duke University Medical Center

Durham, North Carolina 27710

United States

Brody School of Medicine at East Carolina University

Greenville, North Carolina 27834

United States

University Hospitals Cleveland Medical Center

Cleveland, Ohio 44106

United States

Ohio State University Hospital East

Columbus, Ohio 43203

United States

INTEGRIS Cancer Institute of Oklahoma

Oklahoma City, Oklahoma 73109

United States

Allgemeines Krankenhaus der Stadt Linz

Linz, 4020

Austria

Ordensklinikum Linz GmbH Elisabethinen

Linz, 4020

Austria

Klinikum Kreuzschwestern Wels

Wels, 4600

Austria

Acibadem City clinic Tokuda hospital

Sofia, 1407

Bulgaria

UMHAT Aleksandrovska

Sofia, 1431

Bulgaria

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

Sofia, 1431

Bulgaria

National Specialized Hospital for Active Treatment of Hematology Diseases

Sofia, 1797

Bulgaria

UMHAT SofiaMed

Sofia, 1797

Bulgaria

University Hospital Prof. Dr. Stoyan Kirkovich AD

Stara Zagora, 6003

Bulgaria

Beijing YouYi Hospital-Beijing Friendship Hospital Capital Medical University

Beijing, 100050

China

Peking University Third Hospital

Beijing, 100191

China

The First Affiliated Hospital Of Bengbu Medical College

Bengbu, 233004

China

The Affiliated Hospital of Chengde Medical University

Chengde, 067020

China

The Affiliated Hospital of Inner Mongolia Medical University

Hohhot, 010050

China

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

Huai'an, 223300

China

Huizhou Central People's Hospital

Huizhou,

China

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

Tianjin, 300020

China

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

Wuhan,

China

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

Wuxi, 214023

China

Affiliated Hospital of Jiangsu University

Zhenjiang, 212001

China

University Hospital Center Zagreb

Zagreb, 10000

Croatia

Fakultni Nemocnice Ostrava

Ostrava, 708 52

Czechia

Fakultni nemocnice Kralovske Vinohrady

Prague, 100 34

Czechia

CHU d'Amiens-Picardie - Hopital SUD

Amiens, 80054

France

Centre Hospitalier Universitaire (CHU) - Hopital Henri Mondor

Créteil, 94010

France

Centre Hospitalier Universitaire Dijon Bourgogne Medecine Interne et Immunologie Clinique

Dijon, 21079

France

University Hospital Carl Gustav Carus

Dresden, 01307

Germany

Universitaetsklinikum Essen (AoR)

Essen, 45147

Germany

Semmelweis University-Internal Medicine and Haematology

Budapest, 1088

Hungary

Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz

Győr, 9024

Hungary

Mater Misericordiae University Hospital

Dublin, D07 R2WY

Ireland

St James's Hospital - Cancer Clinical Trials Office

Dublin, D08 NHY1

Ireland

Azienda ULSS 7 Pedemontana

Bassano del Grappa, 36061

Italy

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

Florence, 50134

Italy

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Milan, 20122

Italy

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Milan, 20122

Italy

ASST Grande Ospedale Metropolitano Niguarda

Milan, 20162

Italy

AO Maggiore Della Carita

Novara, 28100

Italy

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

Ravenna, 48121

Italy

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

Reggio Emilia, 42123

Italy

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

Torino, 10126

Italy

AO Ordine Mauriziano di Torino

Torino, 10128

Italy

Azienda Sanitaria Universitaria Integrata

Trieste, 34125

Italy

ASST Sette Laghi Ospedale di circolo e Fondazione Macchi

Varese, 21100

Italy

Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy

Bydgoszcz, 85-168

Poland

Pratia Onkologia Katowice

Katowice, 40-519

Poland

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

Lodz, 93-513

Poland

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

Lublin, 20-064

Poland

Uniwersytecki Szpital Kliniczny Nr 4 w Lublinie Centrum Innowacyjnych Terapii

Lublin, 20-090

Poland

Szpital Specjalistyczny im. Jedrzeja Sniadeckiego w Nowym Saczu

Nowy Sącz, 33-300

Poland

Wojewodzki Szpital Zespolony im. Ludwika Rydygiera w Toruniu

Torun, 87-100

Poland

Unidade Local de Saude de Braga

Braga, 4710

Portugal

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

Vila Nova de Gaia, 4434-502

Portugal

Spitalul Clinic Coltea, Clinica de Hematologie

Bucharest, 030171

Romania

Spitalul Clinic Colentina

Bucharest, 20125

Romania

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

Sibiu, 550245

Romania

Spitalul Clinic Judetean de Urgenta Targu-Mures

Târgu Mureş, 540136

Romania

Clinical Center of Serbia

Belgrade, 11000

Serbia

University Clinical Centre of Kragujevac

Kragujevac, 34000

Serbia

Clinical Center of Nis

Niš, 18000

Serbia

University Clinical Hospital Center Zemun

Zemun, 11080

Serbia

General Hospital Djordje Joanovic

Zrenjanin, 23101

Serbia

Complejo Hospitalario Universitario A Coruna

A Coruña, 15006

Spain

Instituto Catalan de Oncologia - Hospital Duran i Reynals

L'Hospitalet de Llobregat, 08908

Spain

Hospital Universitario Ramon y Cajal

Madrid, 28034

Spain

Hospital General Universitario Morales Meseguer

Murcia, 30008

Spain

University Hospital Quironsalud Madrid

Pozuelo de Alarcón, 28223

Spain

Hospital Clinico Universitario - University of Valencia

Valencia, 46010

Spain

Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust

Birmingham, B15 2GW

United Kingdom

Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation

Bradford, BD9 6RJ

United Kingdom

University Hospitals Coventry and Warwickshire NHS Trust

Coventry, CV2 2DX

United Kingdom

Glasgow Royal Infirmary - North Glasgow University Hospital Division

Glasgow, G4 0ET

United Kingdom

University Hospitals of Leicester NHS Trust-Leicester Royal Infirmary

Leicester, LE1 5WW

United Kingdom

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

London, E1 2ES

United Kingdom

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

London, W12 0HS

United Kingdom

Royal Cornwall Hospital (RCH)

Truro, TR1 3LJ

United Kingdom