Hereditary Angioedema (HAE)
Conditions
Brief 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).
Interventions
DRUGFirazyr
Participants with Hereditary angioedema (HAE) receiving treatment with Icatibant acetate (Firazyr) as prescribed by their physician following locally approved prescribing information.
Sponsors
Takeda
Shire
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Hereditary angioedema (HAE) participants in Japan who receive FIRAZYR for first time in the real world clinical setting.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events | 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 | 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 | 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 | 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 | 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 | 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. |
Countries
Japan
Participant flow
Recruitment details
Participants took part in the survey at 93 investigative sites in Japan, from 20 Nov 2018 to 29 Jul 2024.
Pre-assignment details
Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care.
Participants by arm
| Arm | Count |
|---|---|
| Icatibant Acetate Subcutaneous Infusion Participants with Hereditary Angioedema (HAE) received Icatibant acetate subcutaneous infusion as part of a routine medical care. | 155 |
| Total | 155 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Protocol Violation | 24 |
Baseline characteristics
| Characteristic | Icatibant Acetate Subcutaneous Infusion | — |
|---|---|---|
| Age, Continuous | 41.6 years STANDARD_DEVIATION 15.3 | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Sex/Gender, Customized Female | 116 Participants | — |
| Sex/Gender, Customized Male | 38 Participants | — |
| Sex/Gender, Customized Unknown | 1 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 155 |
| other Total, other adverse events | 7 / 155 |
| serious Total, serious adverse events | 1 / 155 |
Outcome results
Primary
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.
Time frame: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Icatibant Acetate Subcutaneous Infusion | Number of Participants With Adverse Drug Reaction | 19 Participants |
Primary
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.
Time frame: 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.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Icatibant Acetate Subcutaneous Infusion | Number of Participants With Adverse Events | 22 Participants |
Primary
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.
Time frame: Up to 3 months
Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Icatibant Acetate Subcutaneous Infusion | Time to Complete Resolution of Attack | 780.0 minutes |
Primary
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.
Time frame: Up to 3 months
Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Icatibant Acetate Subcutaneous Infusion | Time to First Symptom Relief | 60.0 minutes |
Primary
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.
Time frame: Up to 3 months
Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Icatibant Acetate Subcutaneous Infusion | Time to Treatment for Attack | 120.0 minutes |
Primary
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.
Time frame: Up to 3 months
Population: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Icatibant Acetate Subcutaneous Infusion | Total Duration of Attack | 1425.0 minutes |