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

• Sponsor: Massachusetts General Hospital (Other)
• Overall Status: Completed
• CT.gov ID: NCT01457430
• Collaborator: Shire Human Genetic Therapies, Inc. (Industry)
• Enrollment: 19
• Locations: 5
• Arm: 1
• Duration (Months): 28
• Patients Per Site: 3.8
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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

Study Design

• Study Type: Interventional
• 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: Dec 1, 2011
• Actual Primary Completion Date: Apr 1, 2014
• Actual Study Completion Date: Apr 1, 2014

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Time to Complete or Near Complete Resolution From Onset of Symptoms [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 [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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• 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

1. 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.

2. Mental and physical condition allowing patients to complete baseline assessment, to self-administer Icatibant and to follow other study procedures.

3. 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	San Diego Veterans Affairs Medical Center	La Jolla	California	United States
2	UCLA - David Geffen School of Medicine	Los Angeles	California	United States
3	Institute for Allergy and Asthma	Wheaton	Maryland	United States	20902
4	Penn State University	Hershey	Pennsylvania	United States	17033
5	AARA Research Center	Dallas	Texas	United States	75231

Sponsors and Collaborators

• Massachusetts General Hospital
• Shire Human Genetic Therapies, Inc.

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Aleena Banerji, MD, Assistant Professor of Medicine, Massachusetts General Hospital
• ClinicalTrials.gov Identifier: NCT01457430
• Other Study ID Numbers: 2011P001768
• First Posted: Oct 24, 2011
• Last Update Posted: Oct 18, 2016
• Last Verified: Oct 1, 2016

• Individual Participant Data (IPD) Sharing Statement: Undecided
• Plan to Share IPD: Undecided
• Additional relevant MeSH terms: Angioedema; Angioedemas, Hereditary; Icatibant

Study Results

Participant Flow

Arm/Group Title	Icatibant
Arm/Group Description	Icatibant: 30 mg subcutaneous dose of Icatibant
Period Title: Overall Study
STARTED	19
COMPLETED	19
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Icatibant
Arm/Group Description	Icatibant: 30 mg subcutaneous dose of Icatibant
Overall Participants	19
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	17 89.5%
>=65 years	2 10.5%
Age (years) [Mean (Full Range) ]
Mean (Full Range) [years]	41
Sex: Female, Male (Count of Participants)
Female	14 73.7%
Male	5 26.3%
Region of Enrollment (participants) [Number]
United States	19 100%

Outcome Measures

1. Primary Outcome

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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Icatibant Treatment With Health Care Provider	Icatibant Treatment by Self Administration
Arm/Group Description	Icatibant: 30 mg subcutaneous dose of Icatibant given to treat an acute attack of HAE by a health care provider.	Icatibant: 30 mg subcutaneous dose of Icatibant given to treat an acute attack of HAE by self administration.
Measure Participants	19	19
Median (Inter-Quartile Range) [minutes]	735	377

2. Secondary Outcome

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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Icatibant Treatment With Health Care Provider	Icatibant Treatment by Self Administration
Arm/Group Description	Icatibant: 30 mg subcutaneous dose of Icatibant given to treat an acute attack of HAE by a health care provider.	Icatibant: 30 mg subcutaneous dose of Icatibant given to treat an acute attack of HAE by self administration.
Measure Participants	19	19
Median (Inter-Quartile Range) [percent change]	-97	-96

Adverse Events

Time Frame	Adverse events were recorded throughout the study.
Adverse Event Reporting Description	Adverse events were assessed out of total attacks, not total participants. There were 78 HAE attacks with data collected and recorded on adverse events.
Arm/Group Title	Icatibant
Arm/Group Description	Open-label study Icatibant: 30 mg subcutaneous dose of Icatibant
All Cause Mortality
	Icatibant
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Icatibant
	Affected / at Risk (%)	# Events
Total	0/78 (0%)
Other (Not Including Serious) Adverse Events
	Icatibant
	Affected / at Risk (%)	# Events
Total	4/78 (5.1%)
Blood and lymphatic system disorders
Fatigue	1/78 (1.3%)	1
Fever	1/78 (1.3%)	1
Gastrointestinal disorders
Nausea	1/78 (1.3%)	1
Nervous system disorders
Dizziness	1/78 (1.3%)	1

Limitations/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.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.