An Open Label Study of the Effects of Eculizumab in CD59 Deficiency

Summary

The investigators have identified patients with CD59 deficiency that suffers from chronic hemolysis and peripheral demyelinating disease. It was shown that complement terminal pathway can cause inflammation in the peripheral nervous system. Complement can greatly increase the immune attack in the nerves. Eculizumab has already been shown to be effective in a rare blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH). Attacks of PNH are also mediated through complement. Therefore, the investigators of this study are investigating whether by 'turning off' complement in CD59 deficiency, further attacks of hemolysis and nerve injury can be avoided and whether the neurological status will ameliorate.

Timeline

Start

2012-02

Primary completion

2013-03

Completion

2013-03

Publications

• Mevorach D, Reiner I, Grau A, Ilan U, Berkun Y, Ta-Shma A, Elpeleg O, Shorer Z, Edvardson S, Tabib A. Therapy with eculizumab for patients with CD59 p.Cys89Tyr mutation. Ann Neurol. 2016 Nov;80(5):708-717. doi: 10.1002/ana.24770. Epub 2016 Sep 19.

Drugs

Indications