Study of Gefurulimab Treatment in Children Aged 6 to Under 18 With Acetylcholine Receptor Positive Generalized Myasthenia Gravis

What this Trial Is About

Researchers are evaluating the pharmacokinetics how the drug moves through the body and pharmacodynamics the drugs effects of gefurulimab in children aged 6 to under 18 years who have generalized myasthenia gravis with acetylcholine receptor antibodies AChR gMG. This open-label, single-arm Phase 3 study aims to understand how gefurulimab behaves in pediatric patients with this condition and to assess its safety and efficacy. Participants will receive a weight-based loading dose of gefurulimab on the first day, followed by weekly maintenance doses for up to 122 weeks. Gefurulimab is given as a combination product via subcutaneous injection. This regimen is designed to monitor the treatments effects over an extended period while capturing safety data. During the study, children will have their blood tested to measure drug concentration and complement component 5 levels from Day 1 through Week 18. Researchers will also monitor adverse events and changes in disease severity using standardized scores through Week 18. Antibody responses to the drug will be tracked through Week 134. Participants will be regularly assessed for safety and efficacy over the study duration, which may last over two years.

PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis