NCT00289211

C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks

LEVP2005-1/Part A: A Double-blind, Placebo-Controlled, Clinical Study to Investigate the Efficacy and Safety of Purified C1 Esterase Inhibitor (Human) for the Treatment of HAE in Acute Attacks

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 83 (actual)
• Sponsor: Shire · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

The study objective was to determine the safety and efficacy of C1INH-nf for the treatment of acute HAE attacks.

Detailed description

Randomized subjects treated for a qualifying attack were eligible to receive rescue dosing with 1,000 U of C1INH-nf if they did not achieve beginning of substantial relief of the defining symptom within 4 hours after initial treatment with blinded study drug, or if at any time the attack progressed to include airway compromise. A second 1,000 U rescue dose was permitted 60 minutes after the initial rescue dose, if necessary. The study design also allowed for administration of open-label C1INH-nf for laryngeal angioedema attacks, which were non-randomizable events due to the presence of or potential for airway compromise (immediate 1,000 U dose of C1INH-nf, repeated after 60 minutes, if necessary). In addition, subjects were eligible to receive open-label C1INH-nf (1,000 U single dose) prior to emergency surgical (non-cosmetic) procedures. A total of 83 subjects were enrolled in the study. Seventy-one (71) subjects experienced qualifying attacks and were randomized to blinded study drug (36 C1INH-nf, 35 placebo); only the 71 randomized subjects were analyzed for efficacy. An additional 12 subjects were never randomized but received open-label C1INH-nf for treatment of laryngeal angioedema and/or prior to emergency surgical procedures. Of the 35 subjects randomized to placebo, 23 also received C1INH-nf (eg, rescue, open-label). In total, 83 subjects received at least 1 dose of study drug and were analyzed for safety; 71 subjects were exposed to C1INH-nf (59 randomized, 12 open-label only) and 12 subjects were exposed only to placebo.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
BIOLOGICAL	C1 esterase inhibitor [human] (C1INH-nf)
DRUG	Placebo (saline)

Timeline

• Start date: 2005-03-14
• Primary completion: 2007-04-13
• Completion: 2007-04-13
• First posted: 2006-02-09
• Last updated: 2021-06-11
• Results posted: 2010-06-02

Locations

37 sites across 1 country: United States