Hereditary Angioedema (HAE)NCT04057131

FIRAZYR General Drug Use-Results Survey (Japan)

An observational study of Hereditary Angioedema (HAE), run by Shire at 18 sites in Japan.

Completed

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

Trial summary

The objectives of this survey are to collect data to report the safety and efficacy of Firazyr (Icatibant acetate) in the post-marketing phase in participants diagnosed with Hereditary Angioedema (HAE). Read more about this study

Who is it for?: Any age
Conditions the study is for: Hereditary Angioedema (HAE)
What is being tested?: Firazyr
How many people: 179 participants
Where: 18 sitesJapan
Study dates: 20 Nov 2018to 29 Jul 2024
Phase: Observational
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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Study population: Hereditary angioedema (HAE) patients in Japan who receive FIRAZYR for first time in the real world clinical setting are eligible for enrollment in this survey.

Who may be able to join

Hereditary angioedema (HAE) participants in Japan who receive FIRAZYR for first time in the real world clinical setting.

Who cannot join

None listed.

Trial locations and contacts

18 sites in 1 country.

City, postcode or hospital

18 sites in 1 country

Japan18 sites

Asahikawa-city
Asahikawa, Hokkaido, 070-0034
Fukagawa-city
Fukagawa, Hokkaido, 074-0006
Kasama-city
Kasama, Ibaraki, 309-1703
Kawagoe-city
Kawagoe, Saitama, 350-8550
Kishiwada-city
Kishiwada, Osaka, 596-0042
Maebashi-city
Maebashi, Gunma, 371-8511
Maizuru-city
Maizuru, Kyoto, 625-8585
Nagoya-city
Nagoya, Aichi-ken, 453-0046
Niigata-city
Niigata, 950-1197
Numazu-city
Numazu, Shizuoka, 410-0302
Rumoi-city
Rumoi, Hokkaido, 077-0011
Sapporo-city
Sapporo, Hokkaido, 002-8072
Shimada-city
Shimada, Shizuoka, 427-8502
Soka-city
Sōka, Saitama, 340-0041
Tachikawa-city
Tachikawa, Tokyo, 190-0014
Takatsuki-city
Takatsuki, Osaka, 569-0096
Toyohashi-city
Toyohashi, Aichi-ken, 441-8570
Yaezu-city
Yaezu, Shizuoka, 425-0088

Other trials in Japan

About this study

Conditions: Hereditary Angioedema (HAE)
Being tested: Firazyr
Medical subject headings: Angioedemas, Hereditary

What participants receive

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

FirazyrDrug
Also called Icatibant acetate
Participants with Hereditary angioedema (HAE) receiving treatment with Icatibant acetate (Firazyr) as prescribed by their physician following locally approved prescribing information.

Study groups in NCT04057131
Study group	Role	What they receive
Firazyr		Participants with Hereditary angioedema (HAE) receiving treatment with Icatibant acetate (Firazyr) as prescribed by their physician following locally approved prescribing information. Receives: Drug: Firazyr

What is being measured

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

Number of Participants With Adverse Events
An adverse event (AE) is any untoward or undesirable medical occurrence in a participant linked in time with the use of a pharmaceutical/ medicinal product. They are not limited to the events with clear causal relationship with treatment with concerned drug. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not related to the medicinal product.
Measured: Baseline up to end of the study (up to approximately 68 months)
Number of Participants With Adverse Drug Reaction
An adverse event (AE) is any untoward or undesirable medical occurrence in a participant linked in time with the use of a pharmaceutical/ medicinal product. They are not limited to the events with clear causal relationship with treatment with concerned drug. Adverse drug reaction refers to AE related to administered drug.
Measured: Baseline up to end of the study (up to approximately 68 months)
Time to Treatment for Attack
Time to treatment for attack defined as the time between the onset of the attack and the first injection of treatment. Time to treatment for attack was assessed and reported.
Measured: Up to 3 months
Time to First Symptom Relief
Time to first symptom relief defined as the time between the first injection of treatment and first symptom relief. Time to first symptom relief was assessed and reported.
Measured: Up to 3 months
Time to Complete Resolution of Attack
Time to complete resolution of attack defined as the time between the first injection of treatment and the complete resolution of all symptoms. Time to complete resolution of attack was assessed and reported.
Measured: Up to 3 months
Total Duration of Attack
Total duration of attack defined as the time between the onset of the attack and the complete resolution of all symptoms. Total duration of attack was assessed and reported.
Measured: Up to 3 months

Timeline

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

20 Nov 201829 Jul 2024

First posted15 Aug 2019
Study start20 Nov 2018
Primary completion29 Jul 2024
Study completion29 Jul 2024

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Hereditary Angioedema (HAE)Studies of the same condition, open and under way.
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: Observational
Observational model: Other
Time perspective: Prospective
Enrolment: 179 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04057131
Sponsor's protocol ID: SHP667-401
Status last verified: Feb 2025
First submitted: 13 Aug 2019
Last updated: 21 Feb 2025
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Publications

Hosono K, Hoshino M. Real-World Safety and Effectiveness of Icatibant in Patients With Hereditary Angioedema (HAE): Post-Marketing Surveillance in Japan. J Dermatol. 2026 Aug 21. doi: 10.1111/1346-8138.70447. Online ahead of print.
PubMed 42630033

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 (HAE) trials

Study at a glance

From the registry record, last updated by the sponsor on 21 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: FIRAZYR General Drug Use-Results Survey (Japan)
ClinicalTrials.gov ID: NCT04057131
Acronym / sponsor ID: SHP667-401
Conditions: Hereditary Angioedema (HAE)
Study type: Observational
Recruitment status: Completed
Enrolment: 179 participants (actual)
Lead sponsor: ShireIndustry (pharma, biotech, devices)
Collaborators: Takeda
Study start: 20 Nov 2018 (actual)
Primary completion: 29 Jul 2024 (actual)
Study completion: 29 Jul 2024 (actual)
First posted: 15 Aug 2019 (actual)
Results: Posted 21 Feb 2025

Clinical summary

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

Study design

Observational model: Other
Time perspective: Prospective
Number of arms: 1

Arm	Type	Receives
Firazyr	–	Drug: FirazyrParticipants with Hereditary angioedema (HAE) receiving treatment with Icatibant acetate (Firazyr) as prescribed by their physician following locally approved prescribing information.

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: Hereditary angioedema (HAE) patients in Japan who receive FIRAZYR for first time in the real world clinical setting are eligible for enrollment in this survey.

Key inclusion criteria (1 listed)

Hereditary angioedema (HAE) participants in Japan who receive FIRAZYR for first time in the real world clinical setting.

Key exclusion criteria (0 listed)

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
FirazyrAlso: Icatibant acetateParticipants with Hereditary angioedema (HAE) receiving treatment with Icatibant acetate (Firazyr) as prescribed by their physician following locally approved prescribing information.	Drug	Firazyr

Primary outcome

Number of Participants With Adverse Events
Time frame: Baseline up to end of the study (up to approximately 68 months)
An adverse event (AE) is any untoward or undesirable medical occurrence in a participant linked in time with the use of a pharmaceutical/ medicinal product. They are not limited to the events with clear causal relationship with treatment with concerned drug. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not related to the medicinal product.
Number of Participants With Adverse Drug Reaction
Time frame: Baseline up to end of the study (up to approximately 68 months)
An adverse event (AE) is any untoward or undesirable medical occurrence in a participant linked in time with the use of a pharmaceutical/ medicinal product. They are not limited to the events with clear causal relationship with treatment with concerned drug. Adverse drug reaction refers to AE related to administered drug.
Time to Treatment for Attack
Time frame: Up to 3 months
Time to treatment for attack defined as the time between the onset of the attack and the first injection of treatment. Time to treatment for attack was assessed and reported.
Time to First Symptom Relief
Time frame: Up to 3 months
Time to first symptom relief defined as the time between the first injection of treatment and first symptom relief. Time to first symptom relief was assessed and reported.
Time to Complete Resolution of Attack
Time frame: Up to 3 months
Time to complete resolution of attack defined as the time between the first injection of treatment and the complete resolution of all symptoms. Time to complete resolution of attack was assessed and reported.
Total Duration of Attack
Time frame: Up to 3 months
Total duration of attack defined as the time between the onset of the attack and the complete resolution of all symptoms. Total duration of attack was assessed and reported.

Results and safety

Results posted to the registry on 21 Feb 2025, 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 Acetate Subcutaneous Infusion	179	155

Primary outcome: Number of Participants With Adverse Events

Time frame: Baseline up to end of the study (up to approximately 68 months). Count of participants. Unit: Participants

Measure	Icatibant Acetate Subcutaneous Infusion n = 155
Value	22

Population analysed: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

Primary outcome: Number of Participants With Adverse Drug Reaction

Time frame: Baseline up to end of the study (up to approximately 68 months). Count of participants. Unit: Participants

Measure	Icatibant Acetate Subcutaneous Infusion n = 155
Value	19

Population analysed: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.

Primary outcome: Time to Treatment for Attack

Time frame: Up to 3 months. Median (Inter-Quartile Range). Unit: minutes

Measure	Icatibant Acetate Subcutaneous Infusion n = 100
Value	120.0 (30.0 to 360.0)

Population analysed: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation.

Primary outcome: Time to First Symptom Relief

Time frame: Up to 3 months. Median (Inter-Quartile Range). Unit: minutes

Measure	Icatibant Acetate Subcutaneous Infusion n = 100
Value	60.0 (30.0 to 450.0)

Population analysed: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation.

Adverse events

Time frame: Baseline up to end of the study (up to approximately 68 months). Other adverse events reported at 4% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Icatibant Acetate Subcutaneous Infusion	1 of 155 (0.6%)	7 of 155 (4.5%)	0 of 155

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2025.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 42630033Hosono K, Hoshino M. Real-World Safety and Effectiveness of Icatibant in Patients With Hereditary Angioedema (HAE): Post-Marketing Surveillance in Japan. J Dermatol. 2026 Aug 21. doi: 10.1111/1346-8138.70447. Online ahead of print.
To obtain more information on the study, click here/on this linkLink provided by the sponsor.
Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Related trials for Hereditary Angioedema (HAE)

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.

Study Director study director

Takeda

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

Questions about this trial

What is this clinical trial studying?

An observational study of Hereditary Angioedema (HAE), run by Shire at 18 sites in Japan.

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 1 inclusion and 0 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 21 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites in Japan. 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.

Are results available for this study?

Yes. Results are posted in the registry (first posted 21 Feb 2025). 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?

Number of Participants With Adverse Events (time frame: Baseline up to end of the study (up to approximately 68 months)). The study lists 6 primary outcomes in all. 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 NCT04057131, which also lists 1 linked publication.

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 21 Feb 2025.