NCT04833894

Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis

Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children 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 – 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. Trial details include: * The maximum trial duration for each individual participant will be approximately 28 weeks * The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)

Conditions

• Generalized Myasthenia Gravis

Interventions

Type	Name	Description
BIOLOGICAL	Efgartigimod IV	Intravenous infusion of Efgartigimod

Timeline

• Start date: 2021-10-26
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2021-04-06
• Last updated: 2026-02-18

Locations

25 sites across 12 countries: United States, Austria, Belgium, Canada, France, Georgia, Germany, Italy, Netherlands, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study