Phase 3
N=125
Safety and Efficacy of Eculizumab in Refractory Generalized Myasthenia Gravis (REGAIN Study)
Refractory Generalized Myasthenia Gravis
Bottom Line
View on ClinicalTrials.gov: NCT01997229 ↗Enrolled (actual)
125
Serious AEs
21.6%
Results posted
Feb 2018
Primary outcome: 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 scores on a scale — p=0.0698
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Eculizumab (Biological); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Feb 2016
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 |
Summary
The purpose of this study is to determine if eculizumab is safe and effective for the treatment of refractory generalized Myasthenia Gravis.
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). 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.