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

This study has been completed.
Sponsor:
Information provided by:
CSL Behring
ClinicalTrials.gov Identifier:
NCT00168103
First received: September 12, 2005
Last updated: February 10, 2011
Last verified: February 2011
  Purpose

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 Intervention Phase
Hereditary Angioedema
Biological: C1 Esterase Inhibitor
Biological: Placebo
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, 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

Resource links provided by NLM:


Further study details as provided by CSL Behring:

Primary Outcome Measures:
  • Time to Start of Relief of Symptoms From HAE Attack [ Time Frame: Up to 24 h after start of study treatment ] [ Designated as safety issue: No ]
    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:
  • Number of Subjects With Worsened Intensity of Clinical HAE Symptoms [ Time Frame: Baseline and between 2 and 4 h after start of study treatment ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]

Enrollment: 126
Study Start Date: June 2005
Study Completion Date: December 2007
Primary Completion Date: October 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00168103

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

Additional Information:
Publications:
Additional 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
Study First Received: September 12, 2005
Results First Received: April 21, 2010
Last Updated: February 10, 2011
Health Authority: United States: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration
Argentina: Ministry of Health
Bulgaria: Ministry of Health
Canada: Health Canada
Czech Republic: State Institute for Drug Control
Hungary: National Institute of Pharmacy
Israel: Ministry of Health
Macedonia: Ministry of Health
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
Romania: National Medicines Agency
Russia: Ministry of Health of the Russian Federation
Spain: Ministry of Health and Consumption
Sweden: Medical Products Agency
United Kingdom: Medicines and Healthcare Products Regulatory Agency

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 C1 Inactivator Proteins
Complement C1 Inhibitor Protein
Complement C1s
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 19, 2014