REGAIN: Study Design
Adapted from Nowak et al.1
aPatients enrolling in the OLE first entered a 4-week blinded induction phase: patients who had received SOLIRIS® during REGAIN received
SOLIRIS® 1200 mg (4 vials) on day 1 and at week 2, and placebo (4 vials) at weeks 1 and 3; patients who had received placebo during REGAIN
received SOLIRIS® 900 mg (3 vials) and placebo on day 1 and at weeks 1, 2, and 3. bPatients were eligible to enroll in the OLE within 2 weeks of
completing REGAIN. cPatients who withdrew or discontinued after receiving any amount of SOLIRIS® were required to complete a safety
follow-up visit 8 weeks after their last SOLIRIS® dose.
- IST use and clinical outcomes were assessed throughout the OLE, during which IST use could be adjusted at investigators' discretion
- At OLE baseline, 98.3% of patients were using at least one IST
Primary Endpointa
MG Activities of Daily Living (MG-ADL): Results at 26 weeks2
MG-ADL measures improvements in the activities that patients do every day, like talking, chewing, and combing their hair.
aPrimary analysis (worst-rank) showed no significant difference between SOLIRIS® and placebo in change in MG-ADL score (p=0.0698). The use
of worst-rank analysis was an important limitation as secondary and sensitivity analyses were inconsistent with this primary endpoint result.
bAnalyzed using a repeated-measures analysis of variance with treatment, visit, and a treatment by visit interaction as fixed factors, and a
randomization stratification variable, baseline score, and immunosuppressive therapy treatment status as covariates. These analyses ignore the
use of rescue medication and the occurrence of other efficacy-related protocol deviations. There were 6 subjects in the SOLIRIS® arm and 12
subjects in the placebo arm that required rescue medication.
Key Secondary Endpointa
Quantitative MG Scoring System: Results at 26 weeks2
aAnalyzed using a repeated-measures analysis of variance with treatment, visit, and a treatment by visit interaction as fixed factors, and a
randomization stratification variable, baseline score, and immunosuppressive therapy treatment status as covariates. These analyses ignore
the use of rescue medication and the occurrence of other efficacy-related protocol deviations. There were 6 subjects in the SOLIRIS arm and 12
subjects in the placebo arm that required rescue medication.
In an open-label extension of REGAIN, clinical improvements were experienced with or without ISTs
SOLIRIS® with IST tapering can be considered to ease patient treatment burden
SOLIRIS® was studied in clinical trials in patients who were anti-acetylcholine receptor (AChR) antibody positive and refractory, defined as failure of treatment with two or more immunosuppressive therapies (ISTs) either in combination or as monotherapy, or failed at least one IST and required chronic plasmapheresis, plasma exchange (PE), or intravenous immunoglobulin (IVIg) to control symptoms. Patients continued to receive standard therapy throughout the pivotal clinical trial.
