Conditions: Hereditary Angioedema
Keywords: Hereditary angioedema HAE C1 esterase inhibitor (human) C1INH-nf
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 26
Healthy volunteers: no

Brief summary:
The study objective was to determine the safety and efficacy of C1INH-nf for the prevention of acute HAE attacks.

Detailed description:
Subjects were given diary cards and instructed to document all HAE attacks on a daily basis. Subjects evaluated their symptoms over the previous 24 hours, noting the severity and duration of swelling at each of 5 locations (abdominal, genitourinary, facial, respiratory \[including laryngeal\], and/or extremity).

The study design also allowed for administration of open-label C1INH-nf (1,000 U of C1INH-nf administered IV \[repeated after 60 minutes, if necessary\] for treatment of laryngeal angioedema or if deemed necessary by the investigator; 1,000 U of C1INH-nf administered IV \[single dose\] prior to emergency surgical procedures).

A total of 26 subjects were enrolled in the study. One subject received open-label C1INH-nf but withdrew prior to randomization. Another subject was randomized but withdrew prior to receiving study drug. Twenty-four (24) subjects were randomized and treated with blinded study drug. In total, 25 subjects received at least 1 dose of study drug and were analyzed for safety; all 25 subjects were exposed to C1INH-nf and 23 subjects were exposed to placebo.

Arms:
arm 1: 1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by matching placebo (saline) administered IV every 3 to 4 days for 12 weeks. arm 2: Matching placebo (saline) administered IV every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by 1,000 U of C1INH-nf administered IV every 3 to 4 days for 12 weeks.

Interventions: intervention 1: C1 esterase inhibitor [human] (C1INH-nf) intervention 2: Placebo (saline)
Intervention descriptions: intervention 1: None intervention 2: None

Protocol document: