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

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.

Timeline

Start

2018-12-28

Primary completion

2022-01-06

Completion

2023-11-06

Outcome

Met primary endpoint

registry analysis (superiority test); Eculizumab; p = 0.0004; Least Square Mean -5.8 (95% CI -8.40 to -3.13); Repeated Measures Model NCT03759366 ↗

release “Soliris showed significant improvement in the primary endpoint of change from baseline in Quantitative Myasthenia Gravis (QMG) total score at week 26” astrazeneca.com ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Eculizumab	Monoclonal antibody	300 mg	Intravenous
Subject	Eculizumab	Monoclonal antibody	600 mg	Intravenous
Subject	Eculizumab	Monoclonal antibody	900 mg	Intravenous
Subject	Eculizumab	Monoclonal antibody	1200 mg	Intravenous

Indications