Evaluating Efgartigimod IV Treatment for Chronic Immune Thrombocytopenia in Patients Aged 12 to Under 18 Years

What this Trial Is About

Researchers are evaluating the correct dose of efgartigimod intravenous infusion IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia ITP, a condition characterized by low platelet counts. This study is a Phase 2 and 3 clinical trial sponsored by argenx, aiming to determine appropriate dosing and monitor effects in this pediatric population. Participants will be randomly assigned in a 21 ratio to receive either efgartigimod IV or a placebo IV during a double-blinded treatment period lasting up to 24 weeks. After this period, all participants will receive efgartigimod IV in an open-label treatment phase for one year, with an option to continue for a second year. Following the treatment phases, participants will enter an approximately 8-week follow-up period without study drug, totaling up to 138 weeks in the study. Throughout the study, participants will undergo various assessments including monitoring serum concentrations of efgartigimod, measuring total IgG levels, tracking platelet counts, and evaluating bleeding incidents using pediatric scoring tools. Researchers will also assess quality of life through questionnaires and monitor adverse events over the course of the trial. The study involves regular visits and evaluations to ensure thorough monitoring of treatment effects and safety.

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