A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)

NCT07194850 · tracked via the Priya Life Science Germany tracker

Sponsor

argenx

Phase

Phase 2/3

Started

2025-10-20

Last updated

2026-09-24

Condition(s) studied

Immune Thrombocytopenia (ITP)ITP - Immune ThrombocytopeniaITPImmune Thrombocytopenic PurpuraImmune Thrombocytopenic Purpura ( ITP )Idiopathic Thrombocytopenic PurpuraIdiopathic Thrombocytopenic Purpura (ITP)

Investigational drug(s) / intervention(s)

Efgartigimod IV →Placebo IV

Efgartigimod IV: Intravenous infusion of efgartigimod

Placebo IV: Intravenous infusion of placebo

Study summary

The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP).

The study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV. At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first year open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second year (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/advancejunior

Eligibility

Sex

ALL

Min age

12 Years

Max age

17 Years

Healthy volunteers

Inclusion Criteria: * Is aged 12 to less than 18 years when completing the informed consent process * Has a documented duration of primary ITP of more than 12 months on the date the informed consent process is complete * Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, IVIg, anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab. * Has documented prior response, defined as 1 platelet count of ≥50 × 10\^9/L to at least 1 of the following ITP treatments: prednisone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin * Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab, or splenectomy * Has documented mean platelet count of less than 30 x10\^9/L Exclusion Criteria: * Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP * Nonimmune thrombocytopenia * ITP-associated critical or severe bleeding * History of hereditary thrombocytopenia

Primary outcome measure(s)

Efgartigimod serum concentrations in the DBTP — Up to 24 weeks
Total IgG levels in the DBTP — Up to 24 weeks

Trial sites (22)

Facility	City	Region	Status
Centre Hospitalier Universitaire de Bordeaux Groupe Hospitalier Pellegrin Hopital des Enfants	Bordeaux	France	Recruiting
Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK)	Berlin	Germany	Recruiting
Universitaetsklinikum Freiburg Kinder - Kinder- und Jugendklinik	Freiburg im Breisgau	Germany	Recruiting
Gaslini Children's Hospital	Genoa	Italy	Recruiting
Fondazione IRCCS San Gerardo dei Tintori	Monza	Italy	Recruiting
Bambino Gesu Children's Hospital	Roma	Italy	Recruiting
A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita	Torino	Italy	Recruiting
Local Incorporated Administrative Agency Osaka City Hospital Organization Osaka City General Hospital	Osaka	Osaka	Recruiting
Vilnius University Hospital Santaros Klinikos, Children's Hospital	Vilnius	Lithuania	Recruiting
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny nr 4	Lodz	Poland	Recruiting
Uniwersytecki Szpital Dzieciecy w Lublinie	Lublin	Poland	Recruiting
Institutul Clinic Fundeni	Bucharest	Romania	Recruiting
Institute Of Mother And Child Healthcare Of Serbia Dr Vukan Cupic	Belgrade	Serbia	Recruiting
University Clinical Centre of Kragujevac	Kragujevac	Serbia	Recruiting
Hospital Universitari Vall d'Hebron	Barcelona	Spain	Recruiting
Hospital Sant Joan de Deu Barcelona	Esplugues de Llobregat	Spain	Recruiting
Hospital Infantil Universitario Nino Jesus (HIUNJS)	Madrid	Spain	Recruiting
Hospital Materno-Infantil Universitario Gregorio Maranon	Madrid	Spain	Recruiting
Hospital Universitario 12 de Octubre	Madrid	Spain	Recruiting
Cardiff and Vale NHS Trust - University Hospital of Wales (UHW)	Cardiff	United Kingdom	Recruiting
The Royal Hospital for Children - Glasgow Health Board	Glasgow	United Kingdom	Recruiting
Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust	Manchester	United Kingdom	Recruiting

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A Clinical Trial to Investigate the Long-term Safety and Tolerability, Efficacy, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of ARGX-117 in Adults With Multifocal Motor Neuropathy Active, not recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07194850 on ClinicalTrials.gov ↗ ← All trials in Germany