NCT03759366

A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia Gravis (gMG)

An Open-Label, Multicenter Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Eculizumab in Pediatric Patients With Refractory Generalized Myasthenia Gravis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Alexion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of eculizumab in the treatment of pediatric refractory gMG based on change from Baseline in the Quantitative Myasthenia Gravis (QMG) score for disease severity.

Detailed description

The study will consist of an up to 4-week Screening Period, 26-week Primary Evaluation Treatment Period, an additional (up to) to 208-week Extension Period, and an 8-week Safety Follow-up Period.

Conditions

• Myasthenia Gravis
• Myasthenia Gravis, Juvenile Form
• Myasthenia Gravis, Generalized

Interventions

Type	Name	Description
DRUG	Eculizumab	Eculizumab will be administered by IV infusion.

Timeline

• Start date: 2018-12-28
• Primary completion: 2022-01-06
• Completion: 2023-11-06
• First posted: 2018-11-30
• Last updated: 2024-12-11
• Results posted: 2022-08-25

Locations

11 sites across 2 countries: United States, Japan

Regulatory

• FDA-regulated drug study