Efficacy, Safety and Tolerability of Icatibant for the Treatment of HAE

NCT01457430 | Completed Phase 4 Results DMC

• 1 other identifier: 2011P001768
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 5

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.

Conditions

Hereditary Angioedema

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Percent Change in VAS Scores

  Percent Change in VAS Score from Baseline to 4 Hours

Study Arms (1)

Icatibant

EXPERIMENTAL

Open-label study

Drug: Icatibant

Interventions

Icatibant DRUG

30 mg subcutaneous dose of Icatibant

Also known as: Firazyr

Icatibant

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females at least 18 years of age at the time of informed consent
• 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
• 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.
• Mental and physical condition allowing patients to complete baseline assessment, to self-administer Icatibant and to follow other study procedures.
• Ability to provide signed written informed consent after all aspects of the study have been explained and discussed with the patient.

You may not qualify if:

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

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Shire Human Genetic Therapies, Inc.: collaborator

Study Sites (5)

San Diego Veterans Affairs Medical Center

La Jolla, California, United States

Location

UCLA - David Geffen School of Medicine

Los Angeles, California, United States

Location

Institute for Allergy and Asthma

Wheaton, Maryland, 20902, United States

Location

Penn State University

Hershey, Pennsylvania, 17033, United States

Location

AARA Research Center

Dallas, Texas, 75231, United States

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

icatibant

Condition Hierarchy (Ancestors)

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

Limitations and Caveats

All adverse events were recorded and assessed out of total attacks, not total participants. There were 78 HAE attacks; 4 adverse events were identified.

Results Point of Contact

• Title: Dr. Aleena Banerji
• Organization: MGH Allergy Associates

abanerji@mgh.harvard.edu

617-726-3850

Study Officials

• Aleena Banerji, M.D.

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, Assistant Professor of Medicine

Study Record Dates

• First Submitted: October 13, 2011
• First Posted: October 24, 2011
• Study Start: December 1, 2011
• Primary Completion: April 1, 2014
• Study Completion: April 1, 2014
• Last Updated: October 18, 2016
• Results First Posted: October 18, 2016

Record last verified: 2016-10

Locations

US (5)