A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).

NCT ID: NCT04188379

Last Updated: 2025-03-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 131 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-12-16
• Study Completion Date: 2022-02-03

Brief Summary

This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants with primary ITP.

Conditions

Primary Immune Thrombocytopenia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

efgartigimod

Patient receiving efgartigimod

Group Type: EXPERIMENTAL

efgartigimod

Intervention Type: BIOLOGICAL

Intravenous infusion of efgartigimod

Placebo

Patients receiving placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Intravenous infusion of placebo

Interventions

efgartigimod

Intravenous infusion of efgartigimod

Intervention Type: BIOLOGICAL

Placebo

Intravenous infusion of placebo

Intervention Type: OTHER

Other Intervention Names

ARGX-113

Eligibility Criteria

Inclusion Criteria

• 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).
• Male or female patient aged ≥18 years.
• Confirmed ITP diagnosis, at least 3 months before randomization and according to the American Society of Hematology Criteria, and no known other etiology for thrombocytopenia.
• Diagnosis supported by a response to a prior ITP therapy (other than thrombopoietin receptor agonists [TPO-RAs]), in the opinion of the investigator.
• Mean platelet count of <30×10E9/L from 2 counts: 1 platelet count collected during the screening period and the predose platelet count on the day of randomization.
• 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.
• Women of childbearing potential must have a negative serum pregnancy test at the screening visit and a negative urine pregnancy test at baseline before trial medication (infusion) can be administered.
• 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.

Exclusion Criteria

• ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
• Use of certain medications before the start of the studies (more details in the protocol)
• 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 non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted at any time.
• Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments.
• History of any major thrombotic or embolic event within 12 months prior to randomization.
• History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
• 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.
• Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV), Hepatitis C virus (HCV), Human immunodeficiency virus (HIV)
• Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITPdespite appropriate treatments which could put the patient at undue risk.
• Patients with known medical history of hypersensitivity to any of the ingredients of the IMP.
• Patients who previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the IMP.
• Pregnant or lactating females. More details in the protocol

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

argenx

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Investigator Site 0010038

Tucson, Arizona, United States

Investigator Site 0010045

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

Investigator Site 0010034

Jacksonville, Florida, United States

Investigator site 0010037

Ocala, Florida, United States

Investigator Site 0010042

Iowa City, Iowa, United States

Investigator Site 0010046

Greenville, North Carolina, United States

Investigator Site 0010049

Cleveland, Ohio, United States

Investigator Site 0010040

Columbus, Ohio, United States

Investigator Site 0010041

Philadelphia, Pennsylvania, United States

Investigator Site 0430004

Linz, , Austria

Investigator Site 0430002

Vienna, , Austria

Investigator Site 0430003

Vienna, , Austria

Investigator Site 0320012

Brasschaat, , Belgium

Investigator Site 0320011

Bruges, , Belgium

Investigator Site 0320015

Leuven, , Belgium

Investigator Site 0320014

Turnhout, , Belgium

Investigator Site 0320020

Verviers, , Belgium

Investigator site 0320002

Yvoir, , Belgium

Investigator Site 3590001

Pleven, , Bulgaria

Investigator Site 3590002

Sofia, , Bulgaria

Investigator Site 4200001

Brno, , Czechia

Investigator Site 4200008

Olomouc, , Czechia

Investigator Site 4200006

Ostrava, , Czechia

Investigator Site 4200007

Prague, , Czechia

Investigator Site 0330019

Clermont-Ferrand, , France

Investigator Site 0330009

Créteil, , France

Investigator Site 0330015

Le Mans, , France

Investigator Site 0330018

Montpellier, , France

Investigator Site 0330008

Pessac, , France

Investigator Site 0330016

Périgueux, , France

Investigator Site 0330017

Rouen, , France

Investigator Site 9950006

Tbilisi, , Georgia

Investigator Site 9950007

Tbilisi, , Georgia

Investigator Site 9950008

Tbilisi, , Georgia

Investigator Site 9950009

Tbilisi, , Georgia

Investigator Site 9950011

Tbilisi, , Georgia

Investigator Site 9950012

Tbilisi, , Georgia

Investigator Site 0490010

Düsseldorf, , Germany

Investigator Site 0490008

Essen, , Germany

Investigator Site 0490012

Giessen, , Germany

Investigator Site 0360004

Budapest, , Hungary

Investigator Site 0360006

Debrecen, , Hungary

Investigator Site 0360015

Győr, , Hungary

Investigator site 0360010

Nyíregyháza, , Hungary

Investigator Site 0360014

Szombathely, , Hungary

Investigator Site 0390012

Campobasso, , Italy

Investigator Site 0390014

Milan, , Italy

Investigator Site 0390020

Monza, , Italy

Investigator Site 0390015

Novara, , Italy

Investigator Site 0390010

Ravenna, , Italy

Investigator Site 0390011

Reggio Calabria, , Italy

Investigator Site 0390018

Reggio Emilia, , Italy

Investigator Site 0390019

Rimini, , Italy

Investigator Site 0390021

Roma, , Italy

Investigator Site 0390009

Siena, , Italy

Investigator Site 0390017

Torino, , Italy

Investigator Site 0390016

Trieste, , Italy

Investigator Site 0810024

Bunkyō City, , Japan

Investigator Site 0810015

Hirakata, , Japan

Investigator Site 0810010

Hiroshima, , Japan

Investigator site 0810017

Iruma, , Japan

Investigator site 0810011

Isehara, , Japan

Investigator Site 0810022

Kashiwa, , Japan

Investigator site 0810018

Maebashi, , Japan

Investigator site 0810020

Minatoku, , Japan

Investigator Site 0810021

Niigata, , Japan

Investigator site 0810014

Sapporo, , Japan

Investigator site 0810016

Shibukawa, , Japan

Investigator Site 0810023

Shimotsuke, , Japan

Investigator Site 0810025

Shinjuku-Ku, , Japan

Investigator Site 0310005

Rotterdam, , Netherlands

Investigator Site 0310007

Rotterdam, , Netherlands

Investigator site 0310006

The Hague, , Netherlands

Investigator Site 0480030

Bialystok, , Poland

Investigator Site 0480015

Brzozów, , Poland

Investigator Site 0480010

Bydgoszcz, , Poland

Investigator Site 0480013

Chorzów, , Poland

Investigator Site 0480012

Gdansk, , Poland

Investigator Site 0480008

Katowice, , Poland

Investigator Site 0480011

Lodz, , Poland

Investigator Site 0480014

Lublin, , Poland

Investigator site 0480026

Nowy Sącz, , Poland

Investigator Site 0480016

Wroclaw, , Poland

Investigator site 0070006

Kaluga, , Russia

Investigator Site 0070007

Petrozavodsk, , Russia

Investigator Site 0070013

Rostov-on-Don, , Russia

Investigator Site 0070015

Syktyvkar, , Russia

Investigator Site 0070012

Tula, , Russia

Investigator site 0070010

Ufa, , Russia

Investigator Site 0340006

Barcelona, , Spain

Investigator Site 0340007

Barcelona, , Spain

Investigator Site 0340030

Burgos, , Spain

Investigator Site 0340009

Madrid, , Spain

Investigator Site 0340014

Madrid, , Spain

Investigator Site 0340015

Madrid, , Spain

Investigator site 0340012

Palma de Mallorca, , Spain

Investigator Site 0340013

Seville, , Spain

Investigator Site 0340004

Valencia, , Spain

Investigator Site 0340011

Valencia, , Spain

Investigator Site 0900002

Adana, , Turkey (Türkiye)

Investigator Site 0900007

Adapazarı, , Turkey (Türkiye)

Investigator Site 0900003

Ankara, , Turkey (Türkiye)

Investigator Site 0900006

Ankara, , Turkey (Türkiye)

Investigator Site 0900008

Ankara, , Turkey (Türkiye)

Investigator Site 0900015

Ankara, , Turkey (Türkiye)

Investigator Site 0900016

Edirne, , Turkey (Türkiye)

Investigator Site 0900013

Istanbul, , Turkey (Türkiye)

Investigator Site 0900004

Izmir, , Turkey (Türkiye)

Investigator Site 0900014

Kocaeli, , Turkey (Türkiye)

Investigator Site 0900018

Malatya, , Turkey (Türkiye)

Investigator Site 0900005

Manisa, , Turkey (Türkiye)

Investigator Site 0900010

Mersin, , Turkey (Türkiye)

Investigator Site 0900009

Samsun, , Turkey (Türkiye)

Investigator Site 0900017

Tekirdağ, , Turkey (Türkiye)

Investigator Site 0900019

Trabzon, , Turkey (Türkiye)

Investigator Site 3800022

Kharkiv, , Ukraine

Investigator site 3800006

Mykolayiv, , Ukraine

Investigator Site 0440008

London, , United Kingdom

Investigator Site 0440010

Rhyl, , United Kingdom

Investigator Site 0440012

Southampton, , United Kingdom

Investigator Site 0440014

Truro, , United Kingdom

Countries

United States; Austria; Belgium; Bulgaria; Czechia; France; Georgia; Germany; Hungary; Italy; Japan; Netherlands; Poland; Russia; Spain; Turkey (Türkiye); Ukraine; United Kingdom

References

Broome CM, McDonald V, Miyakawa Y, Carpenedo M, Kuter DJ, Al-Samkari H, Bussel JB, Godar M, Ayguasanosa J, De Beuf K, Rodeghiero F, Michel M, Newland A; ADVANCE Investigator Study Group. Efficacy and safety of the neonatal Fc receptor inhibitor efgartigimod in adults with primary immune thrombocytopenia (ADVANCE IV): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet. 2023 Nov 4;402(10413):1648-1659. doi: 10.1016/S0140-6736(23)01460-5. Epub 2023 Sep 28.

Reference Type: DERIVED

PMID: 37778358 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2019-002100-41

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ARGX-113-1801

Identifier Type: -

Identifier Source: org_study_id