An Open Label Study of the Effects of Eculizumab in CD59 Deficiency
Status:
Unknown status
Trial end date:
2013-03-01
Target enrollment:
Participant gender:
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.