NCT00168103

Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks

Human Pasteurized C1 Esterase Inhibitor Concentrate (CE1145) in Subjects With Congenital C1-INH Deficiency and Acute Abdominal or Facial HAE Attacks

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 126 (actual)
• Sponsor: CSL Behring · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

HAE is a rare disorder characterized by functional C1 esterase inhibitor deficiency. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of swelling of the larynx. This clinical Phase 2/Phase 3 study was designed to provide clinically relevant data on dosing, efficacy and safety in subjects with HAE.

Detailed description

For each subject, only a single abdominal or facial attack was treated and evaluated. After receiving treatment, subjects were observed for a minimum of 4 hours, after which they could be discharged from the study center if they reported onset of symptom relief. Starting from 4 hours after treatment, subjects who reported insufficient or no symptom relief could receive a second dose of double-blind treatment (called "rescue medication") as follows: C1-INH 20 U/kg bw for subjects initially receiving placebo, C1-INH 10 U/kg bw for subjects initially receiving C1-INH 10 U/kg bw, and placebo for subjects initially receiving C1-INH 20 U/kg bw. The study was defined to be successful if the primary outcome measure and at least one of the secondary outcome measures were met in the comparison between the C1-INH 20 U/kg bw group and the Placebo group.

Conditions

• Hereditary Angioedema

Interventions

Type	Name	Description
BIOLOGICAL	C1 Esterase Inhibitor	Single application of C1-INH administered intravenously by slow injection or infusion at a recommended rate of 4mL/min.
BIOLOGICAL	Placebo	Single application of physiological saline solution equivalent to the volume calculated for subjects in the C1-INH 20 U/kg bw arm.

Timeline

• Start date: 2005-06-01
• Primary completion: 2007-10-01
• Completion: 2007-12-01
• First posted: 2005-09-14
• Last updated: 2015-03-31
• Results posted: 2010-08-24

Locations

36 sites across 15 countries: United States, Argentina, Australia, Bulgaria, Canada, Czechia, Hungary, Israel, North Macedonia, Poland, Romania, Russia, Spain, Sweden, United Kingdom