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Completed

NCT Number: NCT00168103

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

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Key information

Conditions

Hereditary Angioedema Angioedema Angioedemas, Hereditary Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Complement Deficiency Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Immunologic Deficiency Syndromes Primary Immunodeficiency Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Study Site, Buenos Aires, Argentina

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

C1 Esterase Inhibitor

Biological

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

Biological

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

Primary outcomes

  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 outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

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

Important dates

Study start
2005
Primary completion
2007
Study completion
2007
First posted
Sep 14, 2005
Registry last updated
Mar 31, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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