NCT07194850From ClinicalTrials.govRecruiting

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

A Multicenter, Randomized, Double-blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITP

Immune Thrombocytopenia (ITP)
ITP - Immune Thrombocytopenia
ITP
Immune Thrombocytopenic Purpura
Immune Thrombocytopenic Purpura ( ITP )
Idiopathic Thrombocytopenic Purpura
Idiopathic Thrombocytopenic Purpura (ITP)

At a glance

Phase: Phase 2
Study type: Interventional
Sponsor: argenx
Enrolment target: 24
Started: 20 October 2025
Main results due: October 2028
Study sites: 22
Registry updated: 24 September 2026

Can you take part?

Ages 12 years to 17 years.
Open to any sex.
You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full eligibility criteria

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

What this study is about

In the sponsor’s own words, from the registry.

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

Study sites(22)

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, Japan
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

Showing 22 of 22 sites. 22 of the 22 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT07194850. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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