ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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

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.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Biological: C1 Esterase Inhibitor Biological: Placebo Phase 2 Phase 3

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 : Interventional  (Clinical Trial)
Actual Enrollment : 126 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Human Pasteurized C1 Esterase Inhibitor Concentrate (CE1145) in Subjects With Congenital C1-INH Deficiency and Acute Abdominal or Facial HAE Attacks
Study Start Date : June 2005
Actual Primary Completion Date : October 2007
Actual Study Completion Date : December 2007


Arm Intervention/treatment
Experimental: C1-INH 10 U/kg bw
10 Units (U)/kg body weight (bw) dose
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

Experimental: C1-INH 20 U/kg bw
20 U/kg bw dose
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

Placebo Comparator: Placebo 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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. Number of Vomiting Episodes [ Time Frame: Within 4 h after start of study treatment ]

Other Outcome Measures:
  1. 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.

  2. Number of Subjects Receiving Rescue Study Medication [ Time Frame: Within 4 h after start of study treatment ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00168103


  Show 36 Study Locations
Sponsors and Collaborators
CSL Behring
Investigators
Study Director: Program Director, Clinical R&D CSL Behring

Additional Information:
Publications of Results:
Other Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Global Head Clinical Research & Development, CSL Behring
ClinicalTrials.gov Identifier: NCT00168103     History of Changes
Other Study ID Numbers: CE1145_3001
2004-001186-17 ( EudraCT Number )
First Posted: September 14, 2005    Key Record Dates
Results First Posted: August 24, 2010
Last Update Posted: March 31, 2015
Last Verified: February 2011

Keywords provided by CSL Behring:
C1 Inhibitor
Hereditary angioedema
Acute HAE attack

Additional relevant MeSH terms:
Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Complement C1s
Complement C1 Inhibitor Protein
Complement C1 Inactivator Proteins
Immunologic Factors
Physiological Effects of Drugs
Complement Inactivating Agents
Immunosuppressive Agents