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

NCT04188379 | Completed Phase 3 Results DMC FDA Drug

• 2 other identifiers: ARGX-113-18012019-002100-41
• Study Type: interventional
• Target: 131
• Locations: 18 countries
• Sites: 121

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

Keywords

immune thrombocytopenia; sustained platelet count response; bleeding events

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Chronic ITP With a Sustained Platelet Count Response Defined as Achieving Platelet Counts of at Least 50×10^9/L for at Least 4 of the 6 Visits Between Week 19 and 24 of the Trial.

  Percentage of participants with chronic ITP with a sustained platelet count response was defined as achieving platelet counts of at least 50 × 10\^9/L for at least 4 of the 6 visits between Week 19 and 24 of the study.

  From Week 19 up to Week 24

Secondary Outcomes (4)

• Extent of Disease Control Defined as the Number of Cumulative Weeks Over the Planned 24-week Treatment Period With Platelet Counts of ≥50×10^9/L in the Chronic ITP Population

  From Week 1 up to Week 24

• Percentage of Participants With a Sustained Platelet Count Response for at Least 4 of the 6 Visits Between Week 19 and 24 of the Study

  From Week 19 up to Week 24

• Incidence and Severity of the WHO-classified Bleeding Events

  From Week 1 to Week 24

• Percentage of Participants in the Overall Population Achieving Platelet Counts of at Least 50×10^9/L for at Least 6 of the 8 Visits Between Week 17 and Week 24

  From Week 17 up to Week 24

Study Arms (2)

efgartigimod

EXPERIMENTAL

Patient receiving efgartigimod

Biological: efgartigimod

Placebo

PLACEBO COMPARATOR

Patients receiving placebo

Other: Placebo

Interventions

efgartigimod BIOLOGICAL

Intravenous infusion of efgartigimod

Also known as: ARGX-113

efgartigimod

Placebo OTHER

Intravenous infusion of placebo

Placebo

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• argenx: lead

Study Sites (121)

Investigator Site 0010038

Tucson, Arizona, 85711, United States

Location

Investigator Site 0010045

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

Location

Investigator Site 0010034

Jacksonville, Florida, 32204, United States

Location

Investigator site 0010037

Ocala, Florida, 34474, United States

Location

Investigator Site 0010042

Iowa City, Iowa, 52242, United States

Location

Investigator Site 0010046

Greenville, North Carolina, 27834, United States

Location

Investigator Site 0010049

Cleveland, Ohio, 44106, United States

Location

Investigator Site 0010040

Columbus, Ohio, 44106, United States

Location

Investigator Site 0010041

Philadelphia, Pennsylvania, 19104, United States

Location

Investigator Site 0430004

Linz, Austria

Location

Investigator Site 0430002

Vienna, Austria

Location

Investigator Site 0430003

Vienna, Austria

Location

Investigator Site 0320012

Brasschaat, Belgium

Location

Investigator Site 0320011

Bruges, Belgium

Location

Investigator Site 0320015

Leuven, Belgium

Location

Investigator Site 0320014

Turnhout, Belgium

Location

Investigator Site 0320020

Verviers, Belgium

Location

Investigator site 0320002

Yvoir, Belgium

Location

Investigator Site 3590001

Pleven, Bulgaria

Location

Investigator Site 3590002

Sofia, Bulgaria

Location

Investigator Site 4200001

Brno, Czechia

Location

Investigator Site 4200008

Olomouc, Czechia

Location

Investigator Site 4200006

Ostrava, Czechia

Location

Investigator Site 4200007

Prague, Czechia

Location

Investigator Site 0330019

Clermont-Ferrand, France

Location

Investigator Site 0330009

Créteil, France

Location

Investigator Site 0330015

Le Mans, France

Location

Investigator Site 0330018

Montpellier, France

Location

Investigator Site 0330008

Pessac, France

Location

Investigator Site 0330016

Périgueux, France

Location

Investigator Site 0330017

Rouen, France

Location

Investigator Site 9950006

Tbilisi, Georgia

Location

Investigator Site 9950007

Tbilisi, Georgia

Location

Investigator Site 9950008

Tbilisi, Georgia

Location

Investigator Site 9950009

Tbilisi, Georgia

Location

Investigator Site 9950011

Tbilisi, Georgia

Location

Investigator Site 9950012

Tbilisi, Georgia

Location

Investigator Site 0490010

Düsseldorf, Germany

Location

Investigator Site 0490008

Essen, Germany

Location

Investigator Site 0490012

Giessen, Germany

Location

Investigator Site 0360004

Budapest, Hungary

Location

Investigator Site 0360006

Debrecen, Hungary

Location

Investigator Site 0360015

Győr, Hungary

Location

Investigator site 0360010

Nyíregyháza, Hungary

Location

Investigator Site 0360014

Szombathely, Hungary

Location

Investigator Site 0390012

Campobasso, Italy

Location

Investigator Site 0390014

Milan, Italy

Location

Investigator Site 0390020

Monza, Italy

Location

Investigator Site 0390015

Novara, Italy

Location

Investigator Site 0390010

Ravenna, Italy

Location

Investigator Site 0390011

Reggio Calabria, Italy

Location

Investigator Site 0390018

Reggio Emilia, Italy

Location

Investigator Site 0390019

Rimini, Italy

Location

Investigator Site 0390021

Roma, Italy

Location

Investigator Site 0390009

Siena, Italy

Location

Investigator Site 0390017

Torino, Italy

Location

Investigator Site 0390016

Trieste, Italy

Location

Investigator Site 0810024

Bunkyō City, Japan

Location

Investigator Site 0810015

Hirakata, Japan

Location

Investigator Site 0810010

Hiroshima, Japan

Location

Investigator site 0810017

Iruma, Japan

Location

Investigator site 0810011

Isehara, Japan

Location

Investigator Site 0810022

Kashiwa, Japan

Location

Investigator site 0810018

Maebashi, Japan

Location

Investigator site 0810020

Minatoku, Japan

Location

Investigator Site 0810021

Niigata, Japan

Location

Investigator site 0810014

Sapporo, Japan

Location

Investigator site 0810016

Shibukawa, Japan

Location

Investigator Site 0810023

Shimotsuke, Japan

Location

Investigator Site 0810025

Shinjuku-Ku, Japan

Location

Investigator Site 0310005

Rotterdam, Netherlands

Location

Investigator Site 0310007

Rotterdam, Netherlands

Location

Investigator site 0310006

The Hague, Netherlands

Location

Investigator Site 0480030

Bialystok, Poland

Location

Investigator Site 0480015

Brzozów, Poland

Location

Investigator Site 0480010

Bydgoszcz, Poland

Location

Investigator Site 0480013

Chorzów, Poland

Location

Investigator Site 0480012

Gdansk, Poland

Location

Investigator Site 0480008

Katowice, Poland

Location

Investigator Site 0480011

Lodz, Poland

Location

Investigator Site 0480014

Lublin, Poland

Location

Investigator site 0480026

Nowy Sącz, Poland

Location

Investigator Site 0480016

Wroclaw, Poland

Location

Investigator site 0070006

Kaluga, Russia

Location

Investigator Site 0070007

Petrozavodsk, Russia

Location

Investigator Site 0070013

Rostov-on-Don, Russia

Location

Investigator Site 0070015

Syktyvkar, Russia

Location

Investigator Site 0070012

Tula, Russia

Location

Investigator site 0070010

Ufa, Russia

Location

Investigator Site 0340006

Barcelona, Spain

Location

Investigator Site 0340007

Barcelona, Spain

Location

Investigator Site 0340030

Burgos, Spain

Location

Investigator Site 0340009

Madrid, Spain

Location

Investigator Site 0340014

Madrid, Spain

Location

Investigator Site 0340015

Madrid, Spain

Location

Investigator site 0340012

Palma de Mallorca, Spain

Location

Investigator Site 0340013

Seville, Spain

Location

Investigator Site 0340004

Valencia, Spain

Location

Investigator Site 0340011

Valencia, Spain

Location

Investigator Site 0900002

Adana, Turkey (Türkiye)

Location

Investigator Site 0900007

Adapazarı, Turkey (Türkiye)

Location

Investigator Site 0900003

Ankara, Turkey (Türkiye)

Location

Investigator Site 0900006

Ankara, Turkey (Türkiye)

Location

Investigator Site 0900008

Ankara, Turkey (Türkiye)

Location

Investigator Site 0900015

Ankara, Turkey (Türkiye)

Location

Investigator Site 0900016

Edirne, Turkey (Türkiye)

Location

Investigator Site 0900013

Istanbul, Turkey (Türkiye)

Location

Investigator Site 0900004

Izmir, Turkey (Türkiye)

Location

Investigator Site 0900014

Kocaeli, Turkey (Türkiye)

Location

Investigator Site 0900018

Malatya, Turkey (Türkiye)

Location

Investigator Site 0900005

Manisa, Turkey (Türkiye)

Location

Investigator Site 0900010

Mersin, Turkey (Türkiye)

Location

Investigator Site 0900009

Samsun, Turkey (Türkiye)

Location

Investigator Site 0900017

Tekirdağ, Turkey (Türkiye)

Location

Investigator Site 0900019

Trabzon, Turkey (Türkiye)

Location

Investigator Site 3800022

Kharkiv, Ukraine

Location

Investigator site 3800006

Mykolayiv, Ukraine

Location

Investigator Site 0440008

London, United Kingdom

Location

Investigator Site 0440010

Rhyl, United Kingdom

Location

Investigator Site 0440012

Southampton, United Kingdom

Location

Investigator Site 0440014

Truro, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 37778358 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

efgartigimod alfa

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

Limitations and Caveats

No limitations reported for the study.

Results Point of Contact

• Title: Regulatory Manager
• Organization: argenx BV

regulatory@argenx.com

+32 9 310 3400

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

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: December 4, 2019
• First Posted: December 5, 2019
• Study Start: December 16, 2019
• Primary Completion: February 3, 2022
• Study Completion: February 3, 2022
• Last Updated: March 13, 2025
• Results First Posted: March 13, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

BU (2); AU (3); US (9); PL (10); RU (6); BE (6); DE (3); GB (4); TU (16); CZ (4); IT (12); HU (5); ES (10); FR (7); GE (6); NL (3); JP (13); UA (2)