Clinical trial NCT01457430: Efficacy, Safety and Tolerability of Icatibant for the Treatment of HAE
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hereditary Angioedema
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Shire Human Genetic Therapies, Inc.
First posted: 2011-10-24
Enrollment: 19
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Time to Complete or Near Complete Resolution From Onset of Symptoms
timeFrame: Time to complete or near complete resolution of symptoms as reported by the patient, an expected average of 8-10 hours
Secondary outcomes:
measure: Percent Change in VAS Scores
timeFrame: Percent Change in VAS Score from Baseline to 4 Hours
Other outcomes:
Locations:
city: La Jolla
country: United States
facility: San Diego Veterans Affairs Medical Center
state: California
city: Los Angeles
country: United States
facility: UCLA - David Geffen School of Medicine
state: California
city: Wheaton
country: United States
facility: Institute for Allergy and Asthma
state: Maryland
city: Hershey
country: United States
facility: Penn State University
state: Pennsylvania
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
Study officials:
affiliation: Massachusetts General Hospital
name: Aleena Banerji, M.D.
role: PRINCIPAL_INVESTIGATOR
References: