Study of Subcutaneous Zilucoplan in Children and Teens with Generalized Myasthenia Gravis Evaluating Safety, Activity, and How the Drug Works in Ages 2 to Under 18

What this Trial Is About

Researchers are evaluating the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity, and activity of the drug zilucoplan in children and adolescents with generalized myasthenia gravis gMG. This study focuses on pediatric participants aged 2 to less than 18 years who test positive for acetylcholine receptor antibodies and meet specific clinical classifications. The aim is to understand how zilucoplan behaves in the body and its effects in this young population with gMG. Participants will receive zilucoplan administered under the skin subcutaneously in a dose determined by their weight. This is an open-label, uncontrolled study where all participants receive the same treatment. The dosing and treatment period are structured to allow assessment at Week 4 Day 29 to monitor drug levels and biological effects. Vaccination against meningococcal infections and appropriate antibiotic prophylaxis are required before starting treatment if vaccination is incomplete. Throughout the study, researchers will monitor participants for plasma concentrations of zilucoplan, changes in immune system markers, and clinical scores related to daily activities and quality of life at Week 4. Safety assessments include tracking adverse events, infections, and antibody responses to the drug up to Week 15. The total study duration extends to the end of 2026, with ongoing evaluations to ensure participant safety and treatment activity.

A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis