Phase 3
Completed N=125
Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
Source: ClinicalTrials.gov NCT01997229 ↗Enrolled (actual)
125
Serious AEs
21.6%
Results posted
Feb 2018
Primary outcomePrimary: Myasthenia Gravis Activities of Daily Living Profile (MG-ADL): Change From Baseline in MG-ADL Total Score at Week 26 by Worst-Rank Analysis of Covariance (ANCOVA) — 56.6; 68.3 scores on a scale — p=0.0698
◆ Published Evidence
Highly cited
808citations · ~90 / year
Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.
Summary
The purpose of this study is to determine if eculizumab is safe and effective for the treatment of refractory generalized Myasthenia Gravis.
Linked Publications (5)
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Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study.
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Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension.
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Concomitant Immunosuppressive Therapy Use in Eculizumab-Treated Adults With Generalized Myasthenia Gravis During the REGAIN Open-Label Extension Study.
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Eculizumab Pharmacokinetics and Pharmacodynamics in Patients With Generalized Myasthenia Gravis.
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Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Myasthenia Gravis Activities of Daily Living Profile (MG-ADL): Change From Baseline in MG-ADL Total Score at Week 26 by Worst-Rank Analysis of Covariance (ANCOVA) |
56.6; 68.3 | 0.0698 |
Eligibility Criteria
Key Inclusion Criteria
- Male or female patients ≥18 years old
- Diagnosis of MG made by the following tests:
- Positive serologic test for anti-AChR Abs as confirmed at screening, and
- One of the following:
- History of abnormal neuromuscular transmission test demonstrated by single-fiber electromyography (SFEMG) or repetitive nerve stimulation, or
- History of positive anticholinesterase test, e.g. edrophonium chloride test, or
- 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:
- Failed treatment with at least two immunosuppressive agents. Or,
- 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)
Data sourced from ClinicalTrials.gov (NCT01997229) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.