Trials / Recruiting

RecruitingNCT07194850

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

Status: Recruiting
Phase: Phase 2 / Phase 3
Study type: Interventional
Enrollment: 24 (estimated)

Sponsor: argenx · Industry
Sex: All
Age: 12 Years – 17 Years
Healthy volunteers: Not accepted

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

Conditions

Interventions

Type	Name	Description
BIOLOGICAL	Efgartigimod IV	Intravenous infusion of efgartigimod
OTHER	Placebo IV	Intravenous infusion of placebo

Timeline

Start date: 2025-10-20
Primary completion: 2028-10-01
Completion: 2030-10-01
First posted: 2025-09-26
Last updated: 2026-03-06

Locations

8 sites across 5 countries: Italy, Poland, Romania, Spain, United Kingdom

Regulatory

FDA-regulated drug study

Source: ClinicalTrials.gov record NCT07194850. Inclusion in this directory is not an endorsement.