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14 evidence topics · 5 sources

Objective

Prevention objective

“preventing hereditary angioedema attacks.” (opens the source at this quote in a new tab)

Location and study date

Study sites

“41 sites” (opens the source at this quote in a new tab)

Study design

Parallel-group design

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

Eligibility criteria

Age eligibility

“Patients aged ≥ 12 years” (opens the source at this quote in a new tab)

Treatment

Subcutaneous lanadelumab regimens; placebo comparator

Regimen	Dose and interval
Lower-dose monthly	“150 mg q4wks”
Higher-dose monthly	“300 mg q4wks”
Every two weeks	“300 mg q2wks”

Study outcomes

Rationale for using monthly HAE attack rate as the primary endpoint

Primary clinical endpoint

“number of investigator-confirmed attacks of hereditary angioedema” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Randomized treatment groups

Group	Participants
Lanadelumab 150 mg every four weeks	“28”
Lanadelumab 300 mg every four weeks	“29”
Lanadelumab 300 mg every two weeks	“27”
Placebo	“41”

Description of analysis sets

Primary analysis population and model

“Intent-to-Treat” (opens the source at this quote in a new tab)
“Poisson regression model” (opens the source at this quote in a new tab)

Results

Participant disposition

Study completion

Disposition	Participants, n (%)
Completed	“113 (90.4%)”

Baseline characteristics

Age at baseline

Characteristic	Reported value
Mean age, years	“40.7”

Efficacy results

Mean monthly attack rate over 26 weeks

Treatment	Attacks per month
Lanadelumab 150 mg every four weeks	“0.48”
Lanadelumab 300 mg every four weeks	“0.53”
Lanadelumab 300 mg every two weeks	“0.26”
Placebo	“1.97”

Health-related quality of life and patient-reported outcomes

Participants achieving the AE-QoL minimal clinically important difference

Population	Proportion
All lanadelumab regimens pooled	“70%”
Placebo	“37%”
Lanadelumab 300 mg every two weeks	“81%”

Safety results

Injection-site reactions

Treatment	Participants, n (%)
Lanadelumab 150 mg every four weeks, n=28	“16 (57)”
Lanadelumab 300 mg every four weeks, n=29	“13 (45)”
Lanadelumab 300 mg every two weeks, n=27	“15 (56)”
Placebo, n=41	“14 (34)”

Study limitations

Summary

The randomized groups included 125 participants in total, and 113 (90.4%) completed the study. The 26-week placebo-controlled comparison describes repeated lanadelumab prophylaxis, not a comparison with lonvo-z. Differences between the trials in treatment schedules and observation periods prevent the reported attack rates from establishing comparative superiority.