- Adult patients were randomised to receive either SOLIRIS® (n = 62) or placebo (n = 63) for 26 weeks and were subsequently allowed to enter the open-label extension study2,3
- The primary efficacy endpoint was the change from baseline to week 26 in MG-ADL total score measured by worst-rank ANCOVA value3
Adapted from Muppidi S, et al. Muscle Nerve. 2019;14–24. and Howard JF, et al. Lancet Neurol. 2017;16(12):976–986 (main article and supplementary appendix).2,3
* A total of 126 patients were randomised, 125 of whom received treatment and were analysed.3
† Patients received meningococcal vaccination prior to initiating treatment with SOLIRIS® or received prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination.1
‡ Patients underwent a 4-week induction. In the extension study, a similar induction phase was performed in order to maintain the blinded treatment assignment of REGAIN.2
§ On stable doses prior to screening, defined as: AZA: on drug for ≥ 6 months, on stable dose for ≥ 2 months, MMF, MTX, cyclosporine, tacrolimus, or cyclophosphamide: on drug for ≥ 3 months, on stable dose for ≥ 1 month; oral steroids: on stable dose for ≥ 28 days.3
‖ At the discretion of the investigator.2
healthcare professional
- Primary efficacy endpoint was the change from baseline to week 26 in MG-ADL total score measured by worst-rank ANCOVA value
- Primary endpoint (MG-ADL) did not achieve significance between eculizumab and placebo (least-squares mean rank 56.6 [SEM 4.5] vs 68.3 [4.5]; rank-based treatment difference -11.7, 95% CI -24.3 to 0.96; P = 0.0698
- First secondary endpoint, the change in QMG total score from baseline to week 26, as measured by worst-rank ANCOVA, showed a benefit with eculizumab compared with placebo (nominal P = 0.0129). (3 of 4 prospectively defined sensitivity analyses to validate the primary endpoint of MG-ADL achieved P-value < 0.05). (For QMG, 4 of 4 prospectively defined sensitivity analyses achieved P-value < 0.05)
- Patients who failed treatment for at least one year with 2 or more immunosuppressant therapies (either in combination or as monotherapy), i.e. patients continued to have impairment in activities of daily living despite immunosuppressant therapies1
- Or, patients who failed at least one immunosuppressant therapy and required chronic plasma exchange (PLEX) or intravenous immunoglobulin (IVIg) to control symptoms, i.e., patients require PLEX or IVIg on a regular basis for the management of muscle weakness at least every 3 months over previous 12 months1
Adapted from SOLIRIS® (eculizumab) EU Summary of Product Characteristics and Howard JF, et al. Lancet Neurol. 2017;16(12):976–986.
* Patients receiving a cholinesterase inhibitor must have been on a stable dose for ≥ 2 weeks prior to screening.3
† Chronic PLEX or IVIg is defined as > 4 treatments in a year (at least every 3 months over the previous 12 months).3
‡ Immunosuppressants used prior to enrolment included but were not limited to AZA, MMF, MTX, cyclosporine, tacrolimus, cyclophosphamide, corticosteroids, and rituximab. Use of rituximab within 6 months prior to screening was an exclusion criterion.3
§ More than 80% received other immunosuppressive therapies during the study.3
‖ At randomisation stratification: MGFA class I encompasses ocular muscle weakness only (excluded from REGAIN); classes II, III, or IV refer to mild, moderate, or severe weakness, affecting muscles other than ocular, but may also include ocular muscle weakness of any severity; class a: weakness predominantly affecting limb and/or axial muscles; class b: predominantly affecting oropharyngeal and/or respiratory muscles; class V refers to patients in MG crisis (excluded from REGAIN).3* Immunosuppressants used prior to enrolment included but were not limited to AZA, MMF, MTX, cyclosporine, tacrolimus, cyclophosphamide, corticosteroids, and rituximab. Use of rituximab within 6 months prior to screening was an exclusion criterion.3
† Chronic PLEX or IVIg is defined as > 4 treatments in a year (at least every 3 months over the previous 12 months).3Range: 0–24
Measurements: 8-item outcome measure that reflects ocular, bulbar, respiratory, and limb symptoms and their impact on function
Reported by: Patient
- REGAIN MG-ADL response: A change of ≥ 3 points was considered clinical response*
- Mean total score at baseline (SD): SOLIRIS®: 10.5 (3.1); placebo: 9.9 (2.6)
Range: 0–39
Measurements: 13-item evaluation of ocular, facial, bulbar, gross motor, axial, and respiratory weaknesses
Reported by: Physician
- REGAIN MG-ADL response: A change of ≥ 5 points was considered clinical response†
- Mean total score at baseline (SD): SOLIRIS®: 17.3 (5.1); placebo: 16.9 (5.6)
† The proportion of patients with a ≥ 5-point change in QMG total score from baseline at 26 weeks and no rescue therapy was also a secondary endpoint.3
Range: 0–50
Measurements: 10-item weighted assessments of ocular, muscular, bulbar, and respiratory weakness
Reported by: Physician and patient
- Mean total score at baseline (SD): SOLIRIS®: 20.4 (6.1); placebo: 18.9 (6.0)
Range: 0–60
Measurements: 15 assessments of patient wellbeing and independence
Reported by: Patient
- Mean total score at baseline (SD): SOLIRIS®: 33.3 (12.2); placebo: 30.7 (12.7)