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).
This is a phase III, open label, extension trial of ECU-MG-301(HS-13-00805) to evaluate the safety and efficacy of Eculizumab in subjects with refractory generalized myasthenia gravis (gMG). Eculizumab is a humanized monoclonal antibody that was derived from the murine anti-human C5 antibody m5G1.1. Myasthenia Gravis is a rare, debilitating, acquired autoimmune disease of the neuromuscular junction (NMJ), clinically characterized by weakness and fatigability of skeletal muscle. Since complement activation plays a pivotal role in the pathophysiology of MG, eculizumab, a terminal complement inhibitor, as such may benefit patients who continue to have generalized weakness and bulbar signs and symptoms despite current standard of care.
The primary objective of this trial is to evaluate the long-term safety of eculizumab in subjects with refractory gMG. Subjects who complete the 26-week treatment period (Visit 17) in the ECU-MG-301(HS-13-00805) trial may potentially enter this extension trial. There will be 3 periods in this study, Blind Induction Phase, Open-Label Maintenance Phase and Safety Follow-up Period. To preserve the blinded nature of the ECU-MG-301 trial, all subjects must undergo a blind induction phase and will receive blinded Investigational Product (IP) weekly for four (4) doses. Patients who were randomized to the placebo arm in the ECU-MG-301 trial will receive 4 vials IP (3 vials/900 mg eculizumab plus 1 vial placebo) at Visits 1 to 4. Patients who were randomized to the eculizumab arm in the ECU-MG-301 trial will continue to receive 4 vials IP (1200 mg eculizumab) every two weeks at Visits 1 and 3 and placebo at Visits 2 and 4. All subjects will receive open-label eculizumab (4 vials/1200 mg) every 2 weeks during the open level maintenance phase. On-Trial Rescue Therapy (high dose corticosteroid,plasmapheresis /plasma exchange (PE) or Intravenous Immunoglobulin,) will be allowed if a subject experiences clinical deterioration as defined in this protocol. For a subject who is discontinued from this trial, a follow-up visit for safety evaluation will be required. Primary efficacy endpoint is Safety and tolerability of eculizumab. Secondary Efficacy Endpoints will include the total change of QMG (Quantitative Myasthenia Gravis) score and total change of Myasthenia Gravis Composite (MGC) score. Safety analyses will be performed on the Safety Population and the Extension Safety Population includes all subjects who receive at least 1 dose of IP (eculizumab or Placebo). There is no interim analysis planned for this trial.
Enrolling by invitation | Myasthenia Gravis | Site Unknown
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