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

Recruiting now · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Immune Thrombocytopenia (ITP), ITP - Immune Thrombocytopenia, ITP, Immune Thrombocytopenic Purpura, Immune Thrombocytopenic Purpura ( ITP ), Idiopathic Thrombocytopenic Purpura, Idiopathic Thrombocytopenic Purpura (ITP)

In brief

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

Key facts

Study ID: NCT07194850
Run by: argenx
People needed: 24
Starts: 2025-10-20
Expected to finish: 2030-10-01
Last updated by the study team: 2026-07-09

Who can join

Age: 12 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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

Where it is running

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

Full record on ClinicalTrials.gov

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