RecruitingPhase 2Phase 3NCT07194850

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


Sponsor

argenx

Enrollment

24 participants

Start Date

Oct 20, 2025

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 12 YearsMax Age: 17 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called Efgartigimod IV and Placebo IV for people with itp, itp - immune thrombocytopenia, and other related conditions. The study is currently recruiting participants at 20 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

BIOLOGICALEfgartigimod IV

Intravenous infusion of efgartigimod

OTHERPlacebo IV

Intravenous infusion of placebo


Locations(21)

Centre Hospitalier Universitaire de Bordeaux Groupe Hospitalier Pellegrin Hopital des Enfants

Bordeaux, France

Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK)

Berlin, Germany

Universitaetsklinikum Freiburg Kinder - Kinder- und Jugendklinik

Freiburg im Breisgau, Germany

Gaslini Children's Hospital

Genoa, Italy

Fondazione IRCCS San Gerardo dei Tintori

Monza, Italy

Bambino Gesu Children's Hospital

Roma, Italy

A.O.U. Citta della Salute e della Scienza di Torino - Ospedale Infantile Regina Margherita

Torino, Italy

Vilnius University Hospital Santaros Klinikos, Children's Hospital

Vilnius, Lithuania

Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny nr 4

Lodz, Poland

Uniwersytecki Szpital Dzieciecy w Lublinie

Lublin, Poland

Institutul Clinic Fundeni

Bucharest, Romania

Institute Of Mother And Child Healthcare Of Serbia Dr Vukan Cupic

Belgrade, Serbia

University Clinical Centre of Kragujevac

Kragujevac, Serbia

Hospital Universitari Vall d'Hebron

Barcelona, Spain

Hospital Sant Joan de Deu Barcelona

Esplugues de Llobregat, Spain

Hospital Infantil Universitario Nino Jesus (HIUNJS)

Madrid, Spain

Hospital Materno-Infantil Universitario Gregorio Maranon

Madrid, Spain

Hospital Universitario 12 de Octubre

Madrid, Spain

Cardiff and Vale NHS Trust - University Hospital of Wales (UHW)

Cardiff, United Kingdom

The Royal Hospital for Children - Glasgow Health Board

Glasgow, United Kingdom

Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust

Manchester, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT07194850


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