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

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

Tracking Information

• First Submitted Date: October 13, 2011
• First Posted Date: October 24, 2011
• Results First Submitted Date: August 11, 2016
• Results First Posted Date: October 18, 2016
• Last Update Posted Date: October 18, 2016
• Study Start Date: December 2011
• Actual Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 17, 2016)

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.

• Original Primary Outcome Measures (submitted: October 20, 2011): Time to Complete or Near Complete Resolution From Onset of Symptoms [ Time Frame: Time of complete or near complete improvement of symptoms as reported by the patient ]

Current Secondary Outcome Measures (submitted: October 17, 2016)

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

• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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)
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Hereditary Angioedema

Intervention

Drug: Icatibant

30 mg subcutaneous dose of Icatibant

Other Name: Firazyr

Study Arms

Experimental: Icatibant

Open-label study

Intervention: Drug: Icatibant

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: October 17, 2016): 19
• Original Estimated Enrollment (submitted: October 20, 2011): 30
• Actual Study Completion Date: April 2014
• Actual Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01457430
• Other Study ID Numbers: 2011P001768
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided

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

• Responsible Party: Aleena Banerji, Massachusetts General Hospital
• Study Sponsor: Massachusetts General Hospital
• Collaborators: Shire Human Genetic Therapies, Inc.

Investigators

• Principal Investigator: Aleena Banerji, M.D.; Massachusetts General Hospital

• PRS Account: Massachusetts General Hospital
• Verification Date: October 2016