NCT06607627

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

An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 6 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.

Conditions

• Generalized Myasthenia Gravis
• gMG

Interventions

Type	Name	Description
COMBINATION_PRODUCT	Gefurulimab	Combination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.

Timeline

• Start date: 2024-11-13
• Primary completion: 2026-12-15
• Completion: 2029-01-23
• First posted: 2024-09-23
• Last updated: 2026-04-01

Locations

13 sites across 5 countries: United States, Brazil, Japan, Poland, Taiwan

Regulatory

• FDA-regulated drug study
• FDA-regulated device study