COMPACT

14 evidence topics · 5 sources

Objective

Clinical objective

“clinical efficacy of SC CSL830 in the prophylactic treatment of HAE” (opens the source at this quote in a new tab)

Location and study date

Trial locations and global end date

Field	Reported value
Planned region	“Argentina”
Planned region	“Australia”
Planned region	“Canada”
Planned region	“European Union”
Planned region	“United States”
Global end of trial	“2015-10-12”

Study design

Crossover design

“32-week, multicenter, randomized, double-blind, placebo-controlled, crossover study” (opens the source at this quote in a new tab)

Eligibility criteria

Age eligibility

“12 years or older” (opens the source at this quote in a new tab)

Treatment

Subcutaneous regimens and frequency

“60 IU/kg” (opens the source at this quote in a new tab)
“40 IU/kg” (opens the source at this quote in a new tab)
“twice weekly (every 3 or 4 days)” (opens the source at this quote in a new tab)

Study outcomes

Rationale for using HAE attack frequency as the primary endpoint

Primary clinical endpoint

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

Statistical analysis description

Number of participants (Planned and analyzed)

Planned enrollment

Population	Participants
Whole trial	“100”

Actual randomization

Population	Participants
Randomized	“90”

Description of analysis sets

Efficacy evaluation window

“Efficacy was evaluated for the last 14 weeks of each treatment period.” (opens the source at this quote in a new tab)

Results

Participant disposition

Study completion

Disposition	Participants
Completed	“79”

Baseline characteristics

Baseline age

Characteristic	Reported value
Median age, years (range)	“40 (12 to 72)”

Efficacy results

Monthly attack-rate differences versus placebo

Regimen	Mean difference, attacks per month
40 IU/kg twice weekly	“-2.42”
60 IU/kg twice weekly	“-3.51”

Health-related quality of life and patient-reported outcomes

No evidence found.

Safety results

Most common adverse events

“mild and transient local site reactions” (opens the source at this quote in a new tab)

Study limitations

Summary

The crossover study randomized 90 participants, and 79 completed it. Efficacy was evaluated during the last 14 weeks of each treatment period, rather than the entire 32-week study. The findings concern repeated subcutaneous C1 inhibitor prophylaxis versus placebo within a crossover design. They do not establish comparative effectiveness against lonvo-z, which was evaluated in a parallel-group, single-infusion trial.