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

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)

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.

Study Overview

• Status: Completed
• Conditions: Hereditary Angioedema
• Intervention / Treatment: Drug: Icatibant

• Study Type: Interventional
• Enrollment (Actual): 19
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • California
    • La Jolla, California, United States
      • San Diego Veterans Affairs Medical Center
    • Los Angeles, California, United States
      • UCLA - David Geffen School of Medicine
  • Maryland
    • Wheaton, Maryland, United States, 20902
      • Institute for Allergy and Asthma
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State University
  • Texas
    • Dallas, Texas, United States, 75231
      • AARA Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Icatibant; Open-label study	Drug: Icatibant; 30 mg subcutaneous dose of Icatibant; Other Names: Firazyr

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to Complete or Near Complete Resolution From Onset of Symptoms	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.	Time to complete or near complete resolution of symptoms as reported by the patient, an expected average of 8-10 hours

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent Change in VAS Scores	Baseline, 4 hours VAS scale ranges from 0-100 with 0 being the lowest severity and 100 being the highest severity	Percent Change in VAS Score from Baseline to 4 Hours

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Collaborators: Shire Human Genetic Therapies, Inc.
• Investigators: Principal Investigator: Aleena Banerji, M.D., Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start: December 1, 2011
• Primary Completion (Actual): April 1, 2014
• Study Completion (Actual): April 1, 2014

Study Registration Dates

• First Submitted: October 13, 2011
• First Submitted That Met QC Criteria: October 20, 2011
• First Posted (Estimate): October 24, 2011

Study Record Updates

• Last Update Posted (Estimate): October 18, 2016
• Last Update Submitted That Met QC Criteria: October 17, 2016
• Last Verified: October 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Skin Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Hypersensitivity, Immediate; Genetic Diseases, Inborn; Skin Diseases, Vascular; Hypersensitivity; Urticaria; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Angioedema; Angioedemas, Hereditary; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Analgesics; Sensory System Agents; Anti-Inflammatory Agents, Non-Steroidal; Analgesics, Non-Narcotic; Anti-Inflammatory Agents; Antirheumatic Agents; Immunosuppressive Agents; Immunologic Factors; Complement Inactivating Agents; Bradykinin B2 Receptor Antagonists; Bradykinin Receptor Antagonists; Icatibant
• Other Study ID Numbers: 2011P001768

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: The data is under review for publication but individual data will likely not be available