Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)

• Status: Completed
• Trial end date: 2016-06-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to determine if eculizumab is safe and effective for the treatment of refractory generalized Myasthenia Gravis.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals
• Treatments: Eculizumab

Criteria

Key Inclusion Criteria:

• Male or female patients ≥18 years old

• Diagnosis of MG made by the following tests:

1. Positive serologic test for anti-AChR Abs as confirmed at screening, and

2. One of the following:

1. History of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography (SFEMG) or repetitive nerve stimulation, or

2. History of positive anticholinesterase test, e.g. edrophonium chloride test, or

3. Subject has demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician.

• MGFA Clinical Classification Class II to IV at screening.

• MG-ADL total score must be ≥6 at screening and Randomization (Day 1).

• Subjects who have:

1. Failed treatment with at least two immunosuppressive agents. Or,

2. Failed treatment with at least one immunosuppressive agent and require chronic plasma exchange or IVIg

Key Exclusion Criteria:

• History of thymoma or other neoplasms of the thymus

• History of thymectomy within 12 months prior to screening

• MGFA Class I or MG crisis at screening (MGFA Class V)

• Use of rituximab within 6 months prior to screening

• Use of IVIg or PE within 4 weeks prior to Randomization (Day 1)