Hereditary AngioedemaPhase 4IHANCT01457430

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

A Phase 4 study testing Icatibant in people with Hereditary Angioedema, run by Massachusetts General Hospital at 5 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Hereditary Angioedema
What is being tested?: Icatibant
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 19 participants
Where: 5 sitesUnited States
Study dates: Dec 2011to Apr 2014
Phase: Phase 4
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

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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.

Trial locations and contacts

5 sites in 1 country.

City, postcode or hospital

5 sites in 1 country

United States5 sites

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

Other trials in United States

About this study

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)

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
Being tested: Icatibant
Medical subject headings: Angioedemas, Hereditary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

IcatibantDrug
Also called Firazyr
30 mg subcutaneous dose of Icatibant

Study groups in NCT01457430
Study group	Role	What they receive
Icatibant	Experimental	Open-label study Receives: Drug: Icatibant

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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
Baseline, 4 hours VAS scale ranges from 0-100 with 0 being the lowest severity and 100 being the highest severity
Measured: Percent Change in VAS Score from Baseline to 4 Hours

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2011Apr 2014

First posted24 Oct 2011 (estimated)
Study startDec 2011
Primary completionApr 2014
Study completionApr 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Hereditary AngioedemaStudies of the same condition, open and under way.
More trials run by Massachusetts General HospitalThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

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

IDs, dates and publications

ClinicalTrials.gov ID: NCT01457430
Sponsor's protocol ID: 2011P001768
Acronym: IHA
Status last verified: Oct 2016
First submitted: 13 Oct 2011
Last updated: 18 Oct 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: Undecided: The data is under review for publication but individual data will likely not be available

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
Donidalorsen Treatment in Children With Hereditary Angioedema
Hereditary Angioedema (HAE)
RecruitingObservational study
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
Hereditary Angioedema (HAE)
Active, not recruitingPhase 3
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Hereditary Angioedema
RecruitingPhase 3
Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks
Acute Hereditary Angio Edema
Active, not recruitingPhase 1/2
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Hereditary Angioedema

All Hereditary Angioedema trials

Study at a glance

From the registry record, last updated by the sponsor on 18 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

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)
ClinicalTrials.gov ID: NCT01457430
Acronym / sponsor ID: IHA / 2011P001768
Conditions: Hereditary Angioedema
Phase: Phase 4
Study type: Interventional
Recruitment status: Completed
Enrolment: 19 participants (actual)
Lead sponsor: Massachusetts General HospitalUniversity, hospital or other
Collaborators: Shire Human Genetic Therapies, Inc.
Study start: Dec 2011
Primary completion: Apr 2014 (actual)
Study completion: Apr 2014 (actual)
First posted: 24 Oct 2011 (estimated)
Results: Posted 18 Oct 2016

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Icatibant	Experimental	Drug: IcatibantOpen-label study

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

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:
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.

Key exclusion criteria (10 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
IcatibantAlso: Firazyr30 mg subcutaneous dose of Icatibant	Drug	Icatibant

Primary outcome

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.

1 secondary outcome

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

Results and safety

Results posted to the registry on 18 Oct 2016, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Icatibant	19	19

Primary outcome: 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. Median (Inter-Quartile Range). Unit: minutes

Measure	Icatibant Treatment With Health Care Provider n = 19	Icatibant Treatment by Self Administration n = 19
Value	735 (339 to 1563)	377 (220 to 618)

1 secondary outcome is reported in the full results.

Adverse events

Time frame: Adverse events were recorded throughout the study.. Other adverse events reported at 1% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Icatibant	0 of 78 (0%)	4 of 78 (5.1%)	Not reported

Limitations and caveats (from the sponsor): All adverse events were recorded and assessed out of total attacks, not total participants. There were 78 HAE attacks; 4 adverse events were identified.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 18 Oct 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: Undecided. The data is under review for publication but individual data will likely not be available

Related trials for Hereditary Angioedema

RecruitingPhase 3
Donidalorsen Treatment in Children With Hereditary Angioedema
Hereditary Angioedema (HAE)
RecruitingObservational study
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
Hereditary Angioedema (HAE)
Active, not recruitingPhase 3
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
Hereditary Angioedema
RecruitingPhase 3
Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks
Acute Hereditary Angio Edema
Active, not recruitingPhase 1/2
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
Hereditary Angioedema

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Aleena Banerji, M.D. principal investigator

Massachusetts General Hospital

5 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Icatibant in people with Hereditary Angioedema, run by Massachusetts General Hospital at 5 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Oct 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01457430.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Oct 2016.