EXPAND
Study design
EXPAND Phase 3 study design: Event-driven design allowed for adequate observation period for the assessment of efficacy and safety while reducing exposure to placebo1
![EXPAND – Study Design](/sites/siponimodinfo_com/files/styles/oneup_layout_desktop_2220/public/2021-02/EXPAND%20%E2%80%93%20Study%20Design-01.jpg?itok=7AHJ--q_)
*Open-label part starts when a patient has an 'event'. **Treatment was initiated with a 6-day titration period (0.25 mg-2 mg) with the first maintenance dose of 2 mg reached on Day 6.
The Phase 3 EXPAND study was a randomized, parallel-group, double-blind, placebo-controlled, event-driven, and exposure-driven core part (up to 3 years) followed by an open-label extension part (up to 7 years), which collected long-term data to evaluate the efficacy and safety of siponimod, for up to 10 years. Patients (aged 18–60 years) with SPMS and EDSS score of 3.0–6.5 were randomly assigned (2:1) to receive once-daily oral siponimod 2 mg or placebo for up to 3 years or until the occurrence of a prespecified number of CDP events. The primary endpoint was time to 3-month CDP. CDP was defined as a 1-point increase in EDSS if the baseline score was 3.0–5.0, or a 0.5-point increase if the baseline score was 5.5–6.5, confirmed at a scheduled visit at least 3 months later. The two key secondary endpoints were time to 3-month CDP of at least 20% from baseline in the T25FW test and change from baseline in T2 lesion volume1
- 374 events of 3mCDP and ≥1 year of exposure for >95% randomized patients were required
- Patients with 6mCDP had an option to switch to open-label siponimod or other DMTs while remaining in the core part of the study
- At the EoCP, patients had the option to enter the still ongoing open-label extension part of the trial and receive siponimod for up to additional 7 years
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EXPAND population: Patient demographics and baseline disease characteristics1
![EXPAND population Image](/sites/siponimodinfo_com/files/styles/oneup_layout_desktop_2220/public/2021-02/expand-study-design-1new.png?itok=EGTZxItm)
Data are presented as mean±SD, unless otherwise specified
^Information on the number of relapses in the past 2 years was not available for three patients in the siponimod group and one patient in the placebo group
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EXPAND SPMS population and Phase 3 RMS trials: Patient demographics and baseline
disease characteristics1‒7
![Study](/sites/siponimodinfo_com/files/styles/oneup_layout_desktop_2220/public/2021-03/study_new.png?itok=Dwo5qpwF)
As there are no head-to-head trials of siponimod versus these agents, no comparisons could be made due to differences in study design
#At 2 years;##At 96 weeks; ###At 12 months; §placebo comparator; ¶glatiramer acetate comparator; ǁIFN β-1a comparator; ɭincludes data of patients who received ocrelizumab or ozanimod; ʄtwice daily dimethyl fumarate arm only; ‡alemtuzumab 12 mg arm only; ʆtime since diagnosis; ¥based on inclusion criteria; ƔEDSS score higher than 5.0; ĬMRI cohort
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