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

NCT06544499 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: ARGX-113-24022024-515451-38-00
• Study Type: interventional
• Target: 69
• Locations: 17 countries
• Sites: 94

Brief 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

Conditions

Primary Immune Thrombocytopenia (ITP)

Outcome Measures

Primary Outcomes (1)

• Extent of disease control, defined as the number of cumulative weeks during the 24-week Double-Blinded Treatment Period with platelet counts of at least 50 × 10^9/L

  Up to 24 weeks

Secondary Outcomes (28)

• Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 4 of the 6 study visits between study weeks 19 and 24 of the DBTP

  Up to 6 weeks

• Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 study visits between study weeks 17 and 24 of the DBTP

  Up to 8 weeks

• Proportion of participants achieving a platelet counts of at least 50 × 10^9/L for at least 8 of the 12 study visits between weeks 13 and 24 of the DBTP

  Up to 12 weeks

• Proportion of participants achieving a platelet count of at least 50 × 10^9/L on at least 4 occasions at any time until study week 12 during the DBTP

  Up to 12 weeks

• Time to response, defined as the time to achieve 2 consecutive platelet counts of at least 50 × 10^9/L at any time during the DBTP

  up to 24 weeks

Study Arms (2)

Efgartigimod IV

EXPERIMENTAL

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

Biological: Efgartigimod IV

Placebo IV

EXPERIMENTAL

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

Biological: Efgartigimod IV; Other: Placebo IV

Interventions

Efgartigimod IV BIOLOGICAL

Intravenous infusion of efgartigimod

Efgartigimod IV; Placebo IV

Placebo IV OTHER

Intravenous infusion of placebo

Placebo IV

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• argenx: lead

Study Sites (94)

Mayo Clinic Hospital Scottsdale

Phoenix, Arizona, 85054, United States

RECRUITING

University of Southern California Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

RECRUITING

Sharp Memorial Hospital

Oceanside, California, 92056, United States

RECRUITING

University of Colorado Hospital

Aurora, Colorado, 80045, United States

RECRUITING

Yale University School of Medicine

New Haven, Connecticut, 06510, United States

RECRUITING

Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States

RECRUITING

The University of Chicago Medicine

Chicago, Illinois, 60637, United States

RECRUITING

The University of Iowa Hospitals & Clinics

Iowa City, Iowa, 52242, United States

RECRUITING

Tulane University

New Orleans, Louisiana, 70112, United States

RECRUITING

Henry Ford Health

Detroit, Michigan, 48202, United States

RECRUITING

University of Minnesota

Minneapolis, Minnesota, 55422, United States

RECRUITING

Regional Cancer Care Associates, LLC (RCCA)

Little Silver, New Jersey, 07739, United States

RECRUITING

Clinical Research Alliance Inc.

Westbury, New York, 11590, United States

TERMINATED

Duke University Medical Center

Durham, North Carolina, 27710, United States

RECRUITING

Brody School of Medicine at East Carolina University

Greenville, North Carolina, 27834, United States

RECRUITING

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

RECRUITING

Ohio State University Hospital East

Columbus, Ohio, 43203, United States

RECRUITING

INTEGRIS Cancer Institute of Oklahoma

Oklahoma City, Oklahoma, 73109, United States

RECRUITING

Allgemeines Krankenhaus der Stadt Linz

Linz, 4020, Austria

RECRUITING

Ordensklinikum Linz GmbH Elisabethinen

Linz, 4020, Austria

RECRUITING

Klinikum Kreuzschwestern Wels

Wels, 4600, Austria

RECRUITING

Acibadem City clinic Tokuda hospital

Sofia, 1407, Bulgaria

RECRUITING

UMHAT Aleksandrovska

Sofia, 1431, Bulgaria

RECRUITING

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

Sofia, 1431, Bulgaria

RECRUITING

National Specialized Hospital for Active Treatment of Hematology Diseases

Sofia, 1797, Bulgaria

RECRUITING

UMHAT SofiaMed

Sofia, 1797, Bulgaria

RECRUITING

University Hospital Prof. Dr. Stoyan Kirkovich AD

Stara Zagora, 6003, Bulgaria

RECRUITING

Beijing YouYi Hospital-Beijing Friendship Hospital Capital Medical University

Beijing, 100050, China

RECRUITING

Peking University Third Hospital

Beijing, 100191, China

RECRUITING

The First Affiliated Hospital Of Bengbu Medical College

Bengbu, 233004, China

RECRUITING

The Affiliated Hospital of Chengde Medical University

Chengde, 067020, China

RECRUITING

The Affiliated Hospital of Inner Mongolia Medical University

Hohhot, 010050, China

RECRUITING

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

Huai'an, 223300, China

RECRUITING

Huizhou Central People's Hospital

Huizhou, China

RECRUITING

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

Tianjin, 300020, China

RECRUITING

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

Wuhan, China

RECRUITING

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

Wuxi, 214023, China

RECRUITING

Affiliated Hospital of Jiangsu University

Zhenjiang, 212001, China

RECRUITING

University Hospital Center Zagreb

Zagreb, 10000, Croatia

RECRUITING

Fakultni Nemocnice Ostrava

Ostrava, 708 52, Czechia

RECRUITING

Fakultni nemocnice Kralovske Vinohrady

Prague, 100 34, Czechia

RECRUITING

CHU d'Amiens-Picardie - Hopital SUD

Amiens, 80054, France

RECRUITING

Centre Hospitalier Universitaire (CHU) - Hopital Henri Mondor

Créteil, 94010, France

RECRUITING

Centre Hospitalier Universitaire Dijon Bourgogne Medecine Interne et Immunologie Clinique

Dijon, 21079, France

RECRUITING

University Hospital Carl Gustav Carus

Dresden, 01307, Germany

RECRUITING

Universitaetsklinikum Essen (AoR)

Essen, 45147, Germany

RECRUITING

Semmelweis University-Internal Medicine and Haematology

Budapest, 1088, Hungary

RECRUITING

Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz

Győr, 9024, Hungary

RECRUITING

Mater Misericordiae University Hospital

Dublin, D07 R2WY, Ireland

RECRUITING

St James's Hospital - Cancer Clinical Trials Office

Dublin, D08 NHY1, Ireland

RECRUITING

Azienda ULSS 7 Pedemontana

Bassano del Grappa, 36061, Italy

RECRUITING

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

Florence, 50134, Italy

RECRUITING

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Milan, 20122, Italy

RECRUITING

Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico

Milan, 20122, Italy

RECRUITING

ASST Grande Ospedale Metropolitano Niguarda

Milan, 20162, Italy

RECRUITING

AO Maggiore Della Carita

Novara, 28100, Italy

RECRUITING

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

Ravenna, 48121, Italy

RECRUITING

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

Reggio Emilia, 42123, Italy

RECRUITING

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

Torino, 10126, Italy

RECRUITING

AO Ordine Mauriziano di Torino

Torino, 10128, Italy

RECRUITING

Azienda Sanitaria Universitaria Integrata

Trieste, 34125, Italy

RECRUITING

ASST Sette Laghi Ospedale di circolo e Fondazione Macchi

Varese, 21100, Italy

RECRUITING

Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy

Bydgoszcz, 85-168, Poland

RECRUITING

Pratia Onkologia Katowice

Katowice, 40-519, Poland

RECRUITING

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

Lodz, 93-513, Poland

RECRUITING

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

Lublin, 20-064, Poland

RECRUITING

Uniwersytecki Szpital Kliniczny Nr 4 w Lublinie Centrum Innowacyjnych Terapii

Lublin, 20-090, Poland

RECRUITING

Szpital Specjalistyczny im. Jedrzeja Sniadeckiego w Nowym Saczu

Nowy Sącz, 33-300, Poland

RECRUITING

Wojewodzki Szpital Zespolony im. Ludwika Rydygiera w Toruniu

Torun, 87-100, Poland

RECRUITING

Unidade Local de Saude de Braga

Braga, 4710, Portugal

RECRUITING

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

Vila Nova de Gaia, 4434-502, Portugal

RECRUITING

Spitalul Clinic Coltea, Clinica de Hematologie

Bucharest, 030171, Romania

RECRUITING

Spitalul Clinic Colentina

Bucharest, 20125, Romania

RECRUITING

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

Sibiu, 550245, Romania

RECRUITING

Spitalul Clinic Judetean de Urgenta Targu-Mures

Târgu Mureş, 540136, Romania

RECRUITING

Clinical Center of Serbia

Belgrade, 11000, Serbia

RECRUITING

University Clinical Centre of Kragujevac

Kragujevac, 34000, Serbia

RECRUITING

Clinical Center of Nis

Niš, 18000, Serbia

RECRUITING

University Clinical Hospital Center Zemun

Zemun, 11080, Serbia

RECRUITING

General Hospital Djordje Joanovic

Zrenjanin, 23101, Serbia

RECRUITING

Complejo Hospitalario Universitario A Coruna

A Coruña, 15006, Spain

RECRUITING

Instituto Catalan de Oncologia - Hospital Duran i Reynals

L'Hospitalet de Llobregat, 08908, Spain

RECRUITING

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

RECRUITING

Hospital General Universitario Morales Meseguer

Murcia, 30008, Spain

RECRUITING

University Hospital Quironsalud Madrid

Pozuelo de Alarcón, 28223, Spain

RECRUITING

Hospital Clinico Universitario - University of Valencia

Valencia, 46010, Spain

RECRUITING

Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust

Birmingham, B15 2GW, United Kingdom

RECRUITING

Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation

Bradford, BD9 6RJ, United Kingdom

RECRUITING

University Hospitals Coventry and Warwickshire NHS Trust

Coventry, CV2 2DX, United Kingdom

RECRUITING

Glasgow Royal Infirmary - North Glasgow University Hospital Division

Glasgow, G4 0ET, United Kingdom

RECRUITING

University Hospitals of Leicester NHS Trust-Leicester Royal Infirmary

Leicester, LE1 5WW, United Kingdom

RECRUITING

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

London, E1 2ES, United Kingdom

RECRUITING

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

London, W12 0HS, United Kingdom

RECRUITING

Royal Cornwall Hospital (RCH)

Truro, TR1 3LJ, United Kingdom

RECRUITING

Related Links

• Study website

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Central Study Contacts

Sabine Coppieters, MD

CONTACT

857-350-4834; Clinicaltrials@argenx.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 31, 2024
• First Posted: August 9, 2024
• Study Start: October 18, 2024
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): June 1, 2028
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

FR (3); IE (2); AU (3); CN (11); CZ (2); DE (2); BU (6); US (18); SE (5); PL (7); CR (1); PT (2); GB (8); IT (12); ES (6); HU (2); RO (4)