Effisayil 1

19 evidence topics · 5 sources

Objective

Spesolimab versus placebo during an acute GPP flare

“efficacy, safety and tolerability” (opens the source at this quote in a new tab)

Location and study date

Trial dates and geographic scope

Characteristic	Reported value
Start date	“February 20, 2019”
End date	“January 5, 2021”
Countries with enrolling sites	“12”

Study design

Randomized comparator trial

“randomised, double-blind, placebo-controlled” (opens the source at this quote in a new tab)

Eligibility criteria

Permitted age range

“18 to 75 years” (opens the source at this quote in a new tab)

Acute-flare eligibility thresholds

Criterion	Threshold
GPPGA total score	“≥3”
GPPGA pustulation subscore	“≥2”
Body surface area with erythema and pustules	“≥5%”

Treatment

Single intravenous spesolimab dose compared with placebo

Treatment	Dose in mg
Spesolimab	“900”

Study outcomes

Rationale for using GPPGA pustulation subscore as the primary endpoint

Meaning of a pustulation subscore of zero at week 1

“complete pustular clearance” (opens the source at this quote in a new tab)

GPPGA and GPPASI validation

“the GPPGA and GPPASI are valid, reliable and responsive measures for the assessment of GPP disease severity” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Planned enrollment and power

Parameter	Value
Planned participants	“51”
Overall power for the primary and key secondary endpoints simultaneously	“93.9%”

Randomized participants

Population	Participants
Total enrolled	“53”
Spesolimab	“35”
Placebo	“18”

Description of analysis sets

Population for the primary and key secondary endpoints

“intention-to-treat population” (opens the source at this quote in a new tab)

Prespecified endpoint testing

Method	Quoted description
Comparison of response proportions	“Suissa-Shuster Z-pooled test”
Multiplicity control	“hierarchical manner”

Results

Participant disposition

Open-label spesolimab at week 1

Randomized group and measure	Reported value
Spesolimab: participants and percentage receiving another dose	“12 (34%)”
Placebo: participants receiving spesolimab	“15”
Placebo: percentage receiving spesolimab	“(83%)”

Baseline characteristics

Overall trial population

Characteristic	Reported value
Mean age in years	“43”
Female participants	“68%”

Efficacy results

Week 1 response: participants and percentages

Endpoint	Spesolimab	Placebo
Primary: GPPGA pustulation subscore 0	“19 (54)”	“1 (6)”
Key secondary: GPPGA total score 0 or 1	“15 (43)”	“2 (11)”

Week 1 response comparisons

Endpoint	P value
Primary	“<0.001”
Key secondary	“0.02”

Health-related quality of life and patient-reported outcomes

Week 4 symptom and fatigue endpoints after open-label treatment: analysis approach

“reported descriptively” (opens the source at this quote in a new tab)

Safety results

Comparator trial: spesolimab, Effisayil 1

“Among patients assigned to the spesolimab group, infections occurred in 6 of 35 (17%) through the first week” (opens the source at this quote in a new tab)

Study limitations

Summary

The trial enrolled 53 participants. The primary randomized comparison was assessed at week 1; subsequently, 12 participants initially assigned to spesolimab and 15 initially assigned to placebo received open-label spesolimab. This limits interpretation of later outcomes as randomized treatment comparisons. The publication consequently reported later symptom and fatigue outcomes descriptively. This was a placebo-controlled spesolimab study, not a comparison with imsidolimab.