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NCT07287982
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
Conditions: Spinal Muscular Atrophy (SMA)
Sex: All
Ages: 5 Years – 17 Years
Healthy volunteers: No
Phase: PHASE2
Enrollment: 60
Sponsor: argenx
Location: Arkansas Children's Hospital Little Rock Arkansas
Summary
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).
Eligibility Criteria
Inclusion Criteria:
* Is aged ≥5 to \40°) and/or contractures at screening. o History of spinal fusion within 6 months before screening or planned during the study
* Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for daytime treatment while awake. Ventilation used overnight or during daytime naps is acceptable.
Source: ClinicalTrials.gov (NCT07287982). StuddyBuddy aggregates publicly available trial information.