NCT06392386

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

An Open-label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod PH20 SC in Participants From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: argenx · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study. The participants will be in the study for up to 14 weeks.

Conditions

• Generalized Myasthenia Gravis

Interventions

Type	Name	Description
BIOLOGICAL	Efgartigimod PH20 SC	Subcutaneous injections

Timeline

• Start date: 2024-06-28
• Primary completion: 2026-09-30
• Completion: 2026-09-30
• First posted: 2024-04-30
• Last updated: 2025-12-05

Locations

20 sites across 13 countries: United States, Belgium, Canada, Czechia, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study