ECU-MG-302 Phase III Study of Eculizumab in Subjects with GMG
Research type
Research Study
Full title
A phase III, open-label, extension trial of ECU-MG-301 to evaluate the safety and efficacy of eculizumab in subjects with refractory generalized Myasthenia Gravis (gMG)
IRAS ID
159907
Contact name
Saiju Jacob
Contact email
Sponsor organisation
Alexion Pharmaceuticals, Inc.
Eudract number
2013-002191-41
Research summary
The purpose of this study is to determine if eculizumab is safe and effective for the treatment of patients with refractory generalised Myasthenia Gravis (gMG). Eculizumab is a type of drug called a “biologic”, which means that it comes from a living source (such as humans or animals). It has been approved for use in patients with other diseases or disorders and is also being studied for the treatment of further conditions. Myasthenia Gravis (MG) is a rare and chronic neuromuscular disorder characterised by symptoms of weakness and muscle fatigue. The degree of muscle weakness and severity of symptoms varies widely among patients. MG may involve either a single muscle, or a group of muscles (generalised MG {gMG}). There are a variety of treatments available that may reduce symptoms, allowing some patients with MG to live relatively normal lives. However, some patients continue to experience symptoms despite available treatments; these patients are considered to have refractory gMG.
It is expected that a total of approximately 92 patients will be enrolled in ECU-MG-301 study at approximately 100 locations around the world. Since this is an extension to the ECU-MG-301 study, up to approximately 92 subjects may be treated in this extension study at approximately 100 locations worldwide. Eligible participants will be rolled over from ECU-MG-301 study to receive both eculizumab and placebo (looks like eculizumab but does not contain the active ingredient) for the first four weeks. After the first four weeks they will receive eculizumab only. Treatment will be given by intravenous infusion (into the vein). Participants will be closely monitored throughout the study and will be free to withdraw at any time.
REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
14/EM/1137
Date of REC Opinion
27 Oct 2014
REC opinion
Further Information Favourable Opinion