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

March 11, 2015 updated by: CSL Behring

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Enrollment (Actual)

126

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Buenos Aires, Argentina
        • Study Site
  • Australia
      • Westmead, Australia
        • Study Site
  • Bulgaria
      • Plovdiv, Bulgaria
        • Study Site
      • Sofia, Bulgaria
        • Study Site
  • Canada
      • Edmonton, Canada
        • Study Site
      • Ottawa, Canada
        • Study Site
  • Czech Republic
      • Brno, Czech Republic
        • Study Site
  • Hungary
      • Budapest, Hungary
        • Study Site
  • Israel
      • Tel Hashomer, Israel
        • Study Site
  • Macedonia, The Former Yugoslav Republic of
      • Skopje, Macedonia, The Former Yugoslav Republic of
        • Study Site
  • Poland
      • Grodzisk Mazowiecki, Poland
        • Study Site
      • Krakow, Poland
        • Study Site
  • Romania
      • Tirgu-Mures, Romania
        • Study Site
  • Russian Federation
      • Moscow, Russian Federation
        • Study Site 1
      • Moscow, Russian Federation
        • Study Site 2
      • Moscow, Russian Federation
        • Study Site 3
  • Spain
      • Madrid, Spain
        • Study Site
  • Sweden
      • Goeteborg, Sweden
        • Study Site
  • United Kingdom
      • London, United Kingdom
        • Study Site
  • United States
    • California
      • Granada Hills, California, United States, 91344
        • Study Site
    • Florida
      • Weston, Florida, United States, 33331
        • Study Site
    • Georgia
      • Atlanta, Georgia, United States, 30342
        • Study Site
    • Idaho
      • Idaho Falls, Idaho, United States, 83404
        • Study Site
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Study Site
    • Louisiana
      • Shreveport, Louisiana, United States, 71130
        • Study Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Study Site
    • Minnesota
      • Plymouth, Minnesota, United States, 55411
        • Study Site
    • Nebraska
      • Omaha, Nebraska, United States, 68131
        • Study Site
    • New York
      • Bronx, New York, United States, 10461
        • Study Site
    • Ohio
      • Cincinnati, Ohio, United States, 45231
        • Study Site
    • Oklahoma
      • Tulsa, Oklahoma, United States, 74133
        • Study Site
    • Oregon
      • Eugene, Oregon, United States, 97401
        • Study Site
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Study Site
    • South Dakota
      • Rapid City, South Dakota, United States, 57702
        • Study Site
    • Texas
      • Dallas, Texas, United States, 75230
        • Study Site
    • Washington
      • Bellingham, Washington, United States, 98225
        • Study Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (ADULT, OLDER_ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: DOUBLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Names:
  • Physiological saline solution
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Up to 24 h after start of study treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline and between 2 and 4 h after start of study treatment
Number of Vomiting Episodes
Time Frame: Within 4 h after start of study treatment
Within 4 h after start of study treatment

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Up to 24 h after start of study treatment
Number of Subjects Receiving Rescue Study Medication
Time Frame: Within 4 h after start of study treatment
Within 4 h after start of study treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CSL Behring

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2005

Primary Completion (ACTUAL)

October 1, 2007

Study Completion (ACTUAL)

December 1, 2007

Study Registration Dates

First Submitted

September 12, 2005

First Submitted That Met QC Criteria

September 12, 2005

First Posted (ESTIMATE)

September 14, 2005

Study Record Updates

Last Update Posted (ESTIMATE)

March 31, 2015

Last Update Submitted That Met QC Criteria

March 11, 2015

Last Verified

February 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CE1145_3001
  • 2004-001186-17 (EUDRACT_NUMBER)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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