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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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 Summary:

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.


Sponsor: CSL Behring

Current Primary Outcome: Time to Start of Relief of Symptoms From HAE Attack [ Time Frame: Up to 24 h after start of study treatment ]

The start of symptom relief was determined by subject self-assessment. Time to start of symptom relief was set to 24 hours if the subject received rescue medication (blinded study medication, narcotic analgesics, antiemetics, open-label C1-INH, or fresh frozen plasma) at any time point after the start of study treatment but before start of relief.


Original Primary Outcome: Relief of symptoms from hereditary angioedema (HAE) attacks

Current Secondary Outcome:

  • Number of Subjects With Worsened Intensity of Clinical HAE Symptoms [ Time Frame: Baseline and between 2 and 4 h after start of study treatment ]
    Includes any worsening of intensity of at least 1 of the HAE symptoms present at baseline. Routinely checked symptoms included pain, nausea, vomiting, cramps, and diarrhea.
  • Number of Vomiting Episodes [ Time Frame: Within 4 h after start of study treatment ]


Original Secondary Outcome:

  • Proportion of subjects with increased intensity of clinical HAE symptoms between 2 and 4 h after start of study drug administration
  • Number of vomiting episodes within 4 h after start of study treatment


Information By: CSL Behring

Dates:
Date Received: September 12, 2005
Date Started: June 2005
Date Completion:
Last Updated: March 11, 2015
Last Verified: February 2011