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

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

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

Inclusion Criteria6

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 Criteria4

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

Print for Your Doctor

Interested in this trial?

Get notified about updates and connect with the research team.