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

Phase 2

Completed

• Conditions: Hereditary Angioedema
• Interventions: Biological: C1 Esterase Inhibitor; Biological: Placebo

• Registration Number: NCT00168103
• Lead Sponsor: CSL Behring

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 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.

Study Timeline

• Study Start: 2005-06
• Study First Submitted: 2005-09-12
• Study First Submitted QC: 2005-09-12
• Study First Posted: 2005-09-14
• Primary Completion: 2007-10
• Study Completion: 2007-12
• Results First Submitted: 2010-04-21
• Results First Submitted QC: 2010-07-26
• Results First Posted: 2010-08-24
• Status Verified: 2011-02
• Last Update Submitted: 2015-03-11
• Last Update Posted: 2015-03-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 126

Inclusion Criteria

• Documented congenital C1-INH deficiency
• Acute facial or abdominal HAE attack

Key

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Exclusion Criteria

• Acquired angioedema
• Treatment with any other investigational drug within the last 30 days before study entry
• Treatment with any C1-INH concentrate within the previous 7 days

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
C1-INH 10 U/kg bw	C1 Esterase Inhibitor	10 Units (U)/kg body weight (bw) dose
Placebo	Placebo	-
C1-INH 20 U/kg bw	C1 Esterase Inhibitor	20 U/kg bw dose

Primary Outcome Measures

Name	Time	Method
Time to Start of Relief of Symptoms From HAE Attack	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.

Secondary Outcome Measures

Name	Time	Method
Number of Subjects With Worsened Intensity of Clinical HAE Symptoms	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	Within 4 h after start of study treatment

Trial Locations

Locations (4)

Study Site 1; 🇷🇺 Moscow, Russian Federation

Study Site 2; 🇷🇺 Moscow, Russian Federation

Study Site 3; 🇷🇺 Moscow, Russian Federation

Study Site; 🇬🇧 London, United Kingdom