ECULIZUMAB IN MULTIFOCAL MOTOR NEUROPATHY
Research type
Research Study
Full title
Safety and tolerability of Eculizumab in the treatment of multifocal motor neuropathy: a single centre open label study.
IRAS ID
8167
Contact name
Hugh J Willison
Contact email
Sponsor organisation
Greater Glasgow & Clyde Health Board
Eudract number
2008-005748-18
ISRCTN Number
N/A
Clinicaltrials.gov Identifier
N/A
Research summary
Multifocal motor neuropathy (MMN) is a very rare autoimmune nerve disorder characterized by a slowly progressive paralysis of the arms and legs. It is currently treated with a plasma product called intravenous immunoglobulin (IVIg). Patients with MMN show transient improvement with IVIg, but remain disabled with progressive disease and a rather poor long-term outcome. No other forms of treatment show any benefit in MMN. Since MMN is a chronic disease and IVIg has a half life only of about 3 weeks, most patients require regular treatments with IVIg, once every 2-4 weeks for the rest of their life. More effective forms of treatment are required to improve the long-term outcome in patients with MMN. This is why we are proposing to test Eculizumab in MMN.Eculizumab may be of benefit in MMN as follows: patients with MMN have antibodies in their blood that attack the motor nerves. Part of this attack system involves the damaging action of a group of proteins in the blood called the complement system. When antibodies attach to the nerve, they flag the nerve up for complement attack. Eculizumab is a monoclonal antibody that specifically prevents the activation of the complement system by neutralizing one of its key components, a protein called factor C5, rendering complement inactive. The Eculizumab effect in an animal model of antibody and complement mediated nerve damage is very good. Eculizumab is safe and effective in patients with another complement disease affecting red cells called paroxysmal nocturnal haemoglobinuria. It is approved in both the USA and Europe for use in humans. Eculizumab has not yet been used in neurologically diseased patients. This is why the current proposal is for a small pilot study aiming to look especially at safety in MMN patients.
REC name
West of Scotland REC 1
REC reference
08/S0703/164
Date of REC Opinion
4 Dec 2008
REC opinion
Further Information Favourable Opinion