Trial Outcomes & Findings for FIRAZYR General Drug Use-Results Survey (Japan) (NCT NCT04057131)

Results Overview

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.

Recruitment status

COMPLETED

Target enrollment

179 participants

Primary outcome timeframe

Baseline up to end of the study (up to approximately 68 months)

Results posted on

2025-02-21

Participant Flow

Participants took part in the survey at 93 investigative sites in Japan, from 20 Nov 2018 to 29 Jul 2024.

Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.

Participant milestones

Participant milestones
Measure	Icatibant Acetate Subcutaneous Infusion Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Overall Study STARTED	179
Overall Study COMPLETED	155
Overall Study NOT COMPLETED	24

Reasons for withdrawal

Reasons for withdrawal
Measure	Icatibant Acetate Subcutaneous Infusion Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Overall Study Protocol Violation	24

Baseline Characteristics

The number analyzed is the number of participants with data available for analysis.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Icatibant Acetate Subcutaneous Infusion n=155 Participants Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Age, Continuous	41.6 years STANDARD_DEVIATION 15.3 • n=151 Participants • The number analyzed is the number of participants with data available for analysis.
Sex/Gender, Customized Female	116 Participants n=155 Participants
Sex/Gender, Customized Male	38 Participants n=155 Participants
Sex/Gender, Customized Unknown	1 Participants n=155 Participants

PRIMARY outcome

Timeframe: Baseline up to end of the study (up to approximately 68 months)

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

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.

Outcome measures

Outcome measures
Measure	Icatibant Acetate Subcutaneous Infusion n=155 Participants Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Number of Participants With Adverse Events	22 Participants

PRIMARY outcome

Timeframe: Baseline up to end of the study (up to approximately 68 months)

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

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.

Outcome measures

Outcome measures
Measure	Icatibant Acetate Subcutaneous Infusion n=155 Participants Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Number of Participants With Adverse Drug Reaction	19 Participants

PRIMARY outcome

Timeframe: Up to 3 months

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

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.

Outcome measures

Outcome measures
Measure	Icatibant Acetate Subcutaneous Infusion n=100 Participants Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Time to Treatment for Attack	120.0 minutes Interval 30.0 to 360.0

PRIMARY outcome

Timeframe: Up to 3 months

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

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.

Outcome measures

Outcome measures
Measure	Icatibant Acetate Subcutaneous Infusion n=100 Participants Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Time to First Symptom Relief	60.0 minutes Interval 30.0 to 450.0

PRIMARY outcome

Timeframe: Up to 3 months

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

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.

Outcome measures

Outcome measures
Measure	Icatibant Acetate Subcutaneous Infusion n=100 Participants Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Time to Complete Resolution of Attack	780.0 minutes Interval 210.0 to 1800.0

PRIMARY outcome

Timeframe: Up to 3 months

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

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.

Outcome measures

Outcome measures
Measure	Icatibant Acetate Subcutaneous Infusion n=100 Participants Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Total Duration of Attack	1425.0 minutes Interval 420.0 to 2880.0

Adverse Events

Icatibant Acetate Subcutaneous Infusion

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Icatibant Acetate Subcutaneous Infusion n=155 participants at risk Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Injury, poisoning and procedural complications Facial bones fracture	0.65% 1/155 • Baseline up to end of the study (up to approximately 68 months) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
Injury, poisoning and procedural complications Injury	0.65% 1/155 • Baseline up to end of the study (up to approximately 68 months) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.

Other adverse events

Other adverse events
Measure	Icatibant Acetate Subcutaneous Infusion n=155 participants at risk Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
General disorders Injection site pain	4.5% 7/155 • Baseline up to end of the study (up to approximately 68 months) At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.

Additional Information

Study Director

Takeda

Phone: +1-877-825-3327

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place