A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy

This study aims to find the correct dose of ARGX-119 for children with SMA. The study will also look at how safe the study drug is, how well it works, how it moves through the body, and how the immune system responds to it. The study consists of a double-blinded treatment period (DBTP) where participants will either receive ARGX-119 IV or placebo IV, in addition to disease-modifying therapy (DMT) for 24 weeks. Participants who complete the DBTP will enter the open-label active-treatment extension period (ATEP) during which all participants will receive ARGX-119 IV up to 100 weeks (approximately 2 years).

NCT Number	NCT07287982
Lead Sponsor	argenx
Conditions	Spinal Muscular Atrophy (SMA)
Enrollment	60 participants
Start Date	2025-12-19
Primary Completion	2029-05-28 (estimated)
Study Completion	2029-05-28 (estimated)
Updated on ClinicalTrials.gov	2026-08-10