Premium
A randomized, double‐blind, placebo‐controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis
Author(s) -
Howard James F.,
Barohn Richard J.,
Cutter Gary R.,
Freimer Miriam,
Juel Vern C.,
Mozaffar Tahseen,
Mellion Michelle L.,
Benatar Michael G.,
Farrugia Maria Elena,
Wang Jing Jing,
Malhotra Suneil S.,
Kissel John T.
Publication year - 2013
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.23839
Subject(s) - eculizumab , myasthenia gravis , placebo , medicine , refractory (planetary science) , clinical endpoint , crossover study , gastroenterology , complement system , clinical trial , immunology , antibody , pathology , alternative medicine , astrobiology , physics
: Complement activation at the neuromuscular junction is a primary cause of acetylcholine receptor loss and failure of neuromuscular transmission in myasthenia gravis (MG). Eculizumab, a humanized monoclonal antibody, blocks the formation of terminal complement complex by specifically preventing the enzymatic cleavage of complement 5 (C5). Methods : This study was a randomized, double‐blind, placebo‐controlled, crossover trial involving 14 patients with severe, refractory generalized MG (gMG). Results : Six of 7 patients treated with eculizumab for 16 weeks (86%) achieved the primary endpoint of a 3‐point reduction in the quantitative myasthenia gravis (QMG) score. Examining both treatment periods, the overall change in mean QMG total score was significantly different between eculizumab and placebo ( P = 0.0144). After assessing data obtained from all visits, the overall change in mean QMG total score from baseline was found to be significantly different between eculizumab and placebo ( P < 0.0001). Eculizumab was well tolerated. Conclusion : The data suggest that eculizumab may have a role in treating severe, refractory MG. Muscle Nerve, 2013