VANGUARD

13 evidence topics · 4 sources

Objective

Prophylactic treatment objective

“prophylactic treatment for attacks due to hereditary angioedema (HAE).” (opens the source at this quote in a new tab)

Location and study date

Number of trial centers

“28 centers” (opens the source at this quote in a new tab)

Study design

Study design and allocation ratio

“randomised, double-blind, placebo-controlled” (opens the source at this quote in a new tab)
“(3:2)” (opens the source at this quote in a new tab)

Eligibility criteria

Eligibility: age and run-in attacks

“12 years of age and older” (opens the source at this quote in a new tab)
“two or more acute HAE attacks” (opens the source at this quote in a new tab)

Treatment

Administration schedule

“monthly subcutaneous injections of garadacimab” (opens the source at this quote in a new tab)

Study outcomes

Rationale for using monthly HAE attack rate as the primary endpoint

Primary endpoint: six-month observation

“number of HAE attacks per month” (opens the source at this quote in a new tab)

Statistical analysis description

Number of participants (Planned and analyzed)

Randomized and included populations

Population	Participants
Randomized	“65”
Garadacimab included	“39”
Placebo included	“25”

Description of analysis sets

Safety population: minimum treatment exposure

“at least one dose” (opens the source at this quote in a new tab)

Results

Participant disposition

Randomization error

“One patient was randomly assigned in error” (opens the source at this quote in a new tab)

Baseline characteristics

Baseline sex distribution

Characteristic	Participants, n (%)
Female	“38 (59%)”

Efficacy results

Published monthly attack rates and relative treatment effect

Outcome	Reported value
Garadacimab: mean monthly attack rate	“0·27”
Placebo: mean monthly attack rate	“2·01”
Difference in means, percent	“-87%”

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

Nasopharyngitis adverse reactions

Treatment	Participants, n (%)
Garadacimab, n=39	“8 (21)”
Placebo, n=25	“3 (12)”

Study limitations

Summary

Of 65 randomized participants, 64 were included in the reported comparison after one participant was randomized in error. The six-month trial compared monthly garadacimab with placebo, not with lonvo-z. Its group sizes of 39 and 25 participants limit precision for uncommon adverse events. The results should not be used as a head-to-head comparison with a one-time gene-editing treatment.