©2022 Stanford Medicine
Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
Recruiting
Trial ID: NCT01997229
Purpose
The purpose of this study is to determine if eculizumab is safe and effective for the
treatment of refractory generalized Myasthenia Gravis.
Official Title
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Eculizumab in Subjects With Refractory Generalized Myasthenia Gravis (gMG)
Stanford Investigator(s)
Sarada Sakamuri, MD
Clinical Associate Professor, Neurology & Neurological Sciences Clinical Associate Professor (By courtesy), Neurosurgery
Eligibility
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)
Intervention(s):
biological: Eculizumab
drug: Placebo
Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305