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Efficacy, Safety and Tolerability of Icatibant for the Treatment of HAE (IHA)

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. Identifier: NCT01457430
Recruitment Status : Completed
First Posted : October 24, 2011
Results First Posted : October 18, 2016
Last Update Posted : October 18, 2016
Shire Human Genetic Therapies, Inc.
Information provided by (Responsible Party):
Aleena Banerji, Massachusetts General Hospital

Brief Summary:
The investigators propose a study to evaluate the safety, local tolerability, convenience, and efficacy of self-administered Icatibant for the treatment of acute attacks of hereditary angioedema. The investigators believe that self administration with Icatibant for treatment of an acute attack of angioedema will not change the time to complete or near complete resolution of symptoms compared to treatment with Icatibant in a medical facility.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Drug: Icatibant Phase 4

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open Label, Multicenter Study to Evaluate Efficacy, Safety and Tolerability of a Self-Administered Subcutaneous Formulation of Icatibant for the Treatment of Acute Attacks of Hereditary Angioedema (IHA)
Study Start Date : December 2011
Actual Primary Completion Date : April 2014
Actual Study Completion Date : April 2014

Arm Intervention/treatment
Experimental: Icatibant
Open-label study
Drug: Icatibant
30 mg subcutaneous dose of Icatibant
Other Name: Firazyr

Primary Outcome Measures :
  1. Time to Complete or Near Complete Resolution From Onset of Symptoms [ Time Frame: Time to complete or near complete resolution of symptoms as reported by the patient, an expected average of 8-10 hours ]
    Time of onset of HAE attack, time icatibant was administered, and time to complete relief of symptoms were recorded in minutes. Time to complete relief of symptoms was defined as time from onset of symptoms to complete or near complete resolution as reported by the patient.

Secondary Outcome Measures :
  1. Percent Change in VAS Scores [ Time Frame: Percent Change in VAS Score from Baseline to 4 Hours ]
    Baseline, 4 hours VAS scale ranges from 0-100 with 0 being the lowest severity and 100 being the highest severity

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.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Males and females at least 18 years of age at the time of informed consent
  2. Documented diagnosis of hereditary angioedema Type I or II based on ALL of the following criteria:

    • Family and/or medical history
    • Characteristic attack manifestations, recurrent attacks
    • Historical low C4, normal C1q and either low C1-INH or low C1INH function
  3. Women of childbearing potential must use consistently and correctly a highly effective, adequate method of birth control (failure rate less than 1% per year), sexual abstinence or have a vasectomised partner during the duration of the study. Hormonal contraception can be continued if verified by a physician that it doesn't affect the course of hereditary angioedema attacks.
  4. Mental and physical condition allowing patients to complete baseline assessment, to self-administer Icatibant and to follow other study procedures.
  5. Ability to provide signed written informed consent after all aspects of the study have been explained and discussed with the patient.

Exclusion Criteria:

  1. Participation in a clinical therapeutic trial of another investigational medicinal product within the past month (except a previous Icatibant study).
  2. Diagnosis of angioedema other than Type I or Type II hereditary angioedema.
  3. Evidence of symptomatic coronary artery disease based on medical history, in particular, unstable angina pectoris or severe coronary heart disease.
  4. Congestive heart failure (NYHA Class 3 and 4).
  5. Stroke within the past 6 months.
  6. Treatment with angiotensin converting enzyme inhibitor.
  7. Pregnancy and/or breast-feeding.
  8. In the opinion of the investigator: mental condition rendering the patient unable to understand the nature, scope and possible consequences of the study.
  9. In the opinion of the investigator: unlikely to comply with the protocol, for example, uncooperative attitude, inability to return for follow-up visits, or unlikely to complete the study for any reason.
  10. In the opinion of the investigator: inability to complete the patient diary, manage study medication or self-administration of an injection.

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 identifier (NCT number): NCT01457430

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United States, California
San Diego Veterans Affairs Medical Center
La Jolla, California, United States
UCLA - David Geffen School of Medicine
Los Angeles, California, United States
United States, Maryland
Institute for Allergy and Asthma
Wheaton, Maryland, United States, 20902
United States, Pennsylvania
Penn State University
Hershey, Pennsylvania, United States, 17033
United States, Texas
AARA Research Center
Dallas, Texas, United States, 75231
Sponsors and Collaborators
Massachusetts General Hospital
Shire Human Genetic Therapies, Inc.
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Principal Investigator: Aleena Banerji, M.D. Massachusetts General Hospital
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Responsible Party: Aleena Banerji, MD, Assistant Professor of Medicine, Massachusetts General Hospital Identifier: NCT01457430    
Other Study ID Numbers: 2011P001768
First Posted: October 24, 2011    Key Record Dates
Results First Posted: October 18, 2016
Last Update Posted: October 18, 2016
Last Verified: October 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: The data is under review for publication but individual data will likely not be available
Additional relevant MeSH terms:
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Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Immune System Diseases
Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Bradykinin B2 Receptor Antagonists
Bradykinin Receptor Antagonists
Molecular Mechanisms of Pharmacological Action
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors