CompletedNCT01457430IHAUpdated Oct 18, 2016Results posted

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

A Phase 4 interventional study of Icatibant in Hereditary Angioedema, sponsored by Massachusetts General Hospital. Completed at 5 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2016-10-18.

Sponsored by Massachusetts General Hospital · Phase 4, Interventional, and Treatment

Phase: Phase 4
Study type: Interventional
Enrollment: 19
Allocation: Not applicable
Ages: 18 Years and older
Sex: All

Study 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 studied

Hereditary Angioedema

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In context

Angioedema

164 studies on the registry are indexed under Angioedema; 19 are open to participants now.

This study's enrollment of 19 is below the median of 44 across 111 interventional studies indexed under Angioedema.

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Lead sponsor

Massachusetts General Hospital is the lead sponsor of 2,536 studies on the registry; 446 are open to participants now.

Of its 214 completed or terminated interventional studies of FDA-regulated products, 161 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

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.

Exclusion criteria

Exclusion Criteria:

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.

Study design

Phase: Phase 4
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrollment: 19 participants (actual)

Study arms

Experimental
Icatibant
Open-label study
Drug: Icatibant

Interventions

DrugIcatibant
30 mg subcutaneous dose of Icatibant
Also known as: Firazyr

What researchers measure

Primary outcomes

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 frame: Time to complete or near complete resolution of symptoms as reported by the patient, an expected average of 8-10 hours

Secondary outcomes

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
Time frame: Percent Change in VAS Score from Baseline to 4 Hours

Results

Posted Oct 18, 2016

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.

Participant flow

Participant flow — Overall Study
Milestone	Icatibant
Started	19
Completed	19
Not completed	0

Outcome measures

PrimaryTime 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 frame:: Time to complete or near complete resolution of symptoms as reported by the patient, an expected average of 8-10 hours
Reported as:: Median · minutes

Time to Complete or Near Complete Resolution From Onset of Symptoms
minutes	Icatibant Treatment With Health Care Provider	Icatibant Treatment by Self Administration
Time to Complete or Near Complete Resolution From Onset of Symptoms	735 (339 to 1563)	377 (220 to 618)

SecondaryPercent 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

Time frame:: Percent Change in VAS Score from Baseline to 4 Hours
Reported as:: Median · percent change

Percent Change in VAS Scores
percent change	Icatibant Treatment With Health Care Provider	Icatibant Treatment by Self Administration
Percent Change in VAS Scores	-97 (-100 to -73)	-96 (-100 to -80)

Adverse events

Collected over Adverse events were recorded throughout the study.. Non-serious events are listed at a 1% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Icatibant	—	0/78 (0%)	4/78 (5.1%)

Most frequent other events

Most frequent other events
Event	Icatibant
FatigueBlood and lymphatic system disorders	1/78
FeverBlood and lymphatic system disorders	1/78
DizzinessNervous system disorders	1/78
NauseaGastrointestinal disorders	1/78

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)	Icatibant
<=18 years	0
Between 18 and 65 years	17
>=65 years	2

Age, Continuous
Age, Continuous(years)	Icatibant
Mean	41 (19 to 69)

Sex: Female, Male
Sex: Female, Male(Participants)	Icatibant
Female	14
Male	5

Region of Enrollment
Region of Enrollment(participants)	Icatibant
United States	19

Study locations

5 sites

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

References and documents

Individual participant data

Plan to share: Undecided — The data is under review for publication but individual data will likely not be available

No publications or documents are linked to this record.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Oct 18, 2016, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT01457430
Lead sponsor: Massachusetts General Hospital
Collaborators: Shire Human Genetic Therapies, Inc.
Responsible party: Aleena Banerji (MD, Assistant Professor of Medicine, Massachusetts General Hospital) — Principal investigator
First posted: Oct 24, 2011
Start date: Dec 2011
Primary completion: Apr 2014
Completion: Apr 2014
Results posted: Oct 18, 2016
Last update: Oct 18, 2016

Study contacts

Aleena Banerji, M.D.

principal investigator · Massachusetts General Hospital

Oversight

Data monitoring committee: Yes

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Oct 2016. You cannot join it, but the record below documents what was studied.

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