Study of Efgartigimod PH20 SC Injections in Children Aged 2 to Under 18 With Generalized Myasthenia Gravis Evaluating Safety, Dosage, and Effects

What this Trial Is About

Researchers are studying efgartigimod PH20 SC in children aged 2 to less than 18 years who have generalized Myasthenia Gravis gMG. The study aims to understand how the drug is processed by the body, its effects, safety, tolerability, and immune response in this pediatric group. The main goal is to determine the appropriate dose for children using pharmacokinetic and pharmacodynamic data. Participants will receive subcutaneous injections of efgartigimod PH20 SC and be monitored throughout the study, which lasts up to 14 weeks. The study is open-label and uncontrolled, focusing on evaluating the drugs activity and safety. After the follow-up period, eligible participants may join an open-label extension study for continued observation. During the study, researchers will collect blood samples to measure drug levels, immunoglobulin G, and antibodies related to Myasthenia Gravis. They will monitor adverse events, severity of side effects, and changes in disease activity scores suitable for children. Participants will complete assessments of fatigue, quality of life, and global improvement. Safety will be closely observed with regular testing and follow-up visits throughout the study duration.

A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis