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

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ClinicalTrials.gov Identifier: NCT00168103
Recruitment Status : Completed
First Posted : September 14, 2005
Results First Posted : August 24, 2010
Last Update Posted : March 31, 2015
Sponsor:
Information provided by:
CSL Behring

Tracking Information
First Submitted Date  ICMJE September 12, 2005
First Posted Date  ICMJE September 14, 2005
Results First Submitted Date  ICMJE April 21, 2010
Results First Posted Date  ICMJE August 24, 2010
Last Update Posted Date March 31, 2015
Study Start Date  ICMJE June 2005
Actual Primary Completion Date October 2007   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 26, 2010)
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 Measures  ICMJE
 (submitted: September 12, 2005)
Relief of symptoms from hereditary angioedema (HAE) attacks
Change History Complete list of historical versions of study NCT00168103 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 26, 2010)
  • 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 Measures  ICMJE
 (submitted: September 12, 2005)
  • 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
Current Other Pre-specified Outcome Measures
 (submitted: March 11, 2015)
  • Time to Complete Resolution of All HAE Symptoms, Including Pain [ Time Frame: Up to 24 h after start of study treatment ]
    Complete resolution of symptoms was determined by subject self-assessment.
  • Number of Subjects Receiving Rescue Study Medication [ Time Frame: Within 4 h after start of study treatment ]
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks
Official Title  ICMJE 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 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Hereditary Angioedema
Intervention  ICMJE
  • Biological: C1 Esterase Inhibitor
    Single application of C1-INH administered intravenously by slow injection or infusion at a recommended rate of 4mL/min.
    Other Names:
    • Berinert
    • Berinert P
    • CE1145
  • Biological: Placebo
    Single application of physiological saline solution equivalent to the volume calculated for subjects in the C1-INH 20 U/kg bw arm.
    Other Name: Physiological saline solution
Study Arms  ICMJE
  • Experimental: C1-INH 10 U/kg bw
    10 Units (U)/kg body weight (bw) dose
    Intervention: Biological: C1 Esterase Inhibitor
  • Experimental: C1-INH 20 U/kg bw
    20 U/kg bw dose
    Intervention: Biological: C1 Esterase Inhibitor
  • Placebo Comparator: Placebo
    Intervention: Biological: Placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 26, 2010)
126
Original Enrollment  ICMJE
 (submitted: September 12, 2005)
90
Actual Study Completion Date  ICMJE December 2007
Actual Primary Completion Date October 2007   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

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

Key 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Australia,   Bulgaria,   Canada,   Czech Republic,   Hungary,   Israel,   Macedonia, The Former Yugoslav Republic of,   Poland,   Romania,   Russian Federation,   Spain,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00168103
Other Study ID Numbers  ICMJE CE1145_3001
2004-001186-17 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Global Head Clinical Research & Development, CSL Behring
Study Sponsor  ICMJE CSL Behring
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Program Director, Clinical R&D CSL Behring
PRS Account CSL Behring
Verification Date February 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP