Trial Outcomes & Findings for A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema (NCT NCT01386658)

NCT ID: NCT01386658

Last Updated: 2021-06-08

Results Overview

Time to peak concentration (Tmax) of a single SC dose of icatibant was reported.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

32 participants

Primary outcome timeframe

Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Results posted on

2021-06-08

Participant Flow

The study was conducted at 27 study centers in the United States, Germany, Israel, Spain, Argentina, Australia, Austria, Canada, Colombia, Hungary, and Italy between 27 January 2012 (first participant first visit) and 12 March 2018 (last participant last visit).

A total of 32 participants were enrolled and received treatment.

Participant milestones

Participant milestones
Measure
Prepubertal
Participants received a single subcutaneous (SC) injection of 0.4 milligram per kilogram (mg/kg) icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Initial Icatibant Exposure
STARTED
11
21
Initial Icatibant Exposure
COMPLETED
11
9
Initial Icatibant Exposure
NOT COMPLETED
0
12
Icatibant Exposure 2
STARTED
0
9
Icatibant Exposure 2
COMPLETED
0
9
Icatibant Exposure 2
NOT COMPLETED
0
0
Icatibant Exposure 3
STARTED
0
9
Icatibant Exposure 3
COMPLETED
0
9
Icatibant Exposure 3
NOT COMPLETED
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Prepubertal
Participants received a single subcutaneous (SC) injection of 0.4 milligram per kilogram (mg/kg) icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Initial Icatibant Exposure
Withdrawal by Subject
0
9
Initial Icatibant Exposure
Lack of adherence and poor compliance
0
3

Baseline Characteristics

A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal
n=21 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Total
n=32 Participants
Total of all reporting groups
Age, Continuous
8.6 Years
STANDARD_DEVIATION 2.97 • n=93 Participants
14.3 Years
STANDARD_DEVIATION 1.66 • n=4 Participants
12.3 Years
STANDARD_DEVIATION 3.48 • n=27 Participants
Sex: Female, Male
Female
5 Participants
n=93 Participants
8 Participants
n=4 Participants
13 Participants
n=27 Participants
Sex: Female, Male
Male
6 Participants
n=93 Participants
13 Participants
n=4 Participants
19 Participants
n=27 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants
n=93 Participants
2 Participants
n=4 Participants
3 Participants
n=27 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
10 Participants
n=93 Participants
19 Participants
n=4 Participants
29 Participants
n=27 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
Asian
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
White
11 Participants
n=93 Participants
20 Participants
n=4 Participants
31 Participants
n=27 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=93 Participants
0 Participants
n=4 Participants
0 Participants
n=27 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=93 Participants
1 Participants
n=4 Participants
1 Participants
n=27 Participants

PRIMARY outcome

Timeframe: Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Population: Pharmacokinetic (PK) population consisted of participants who were treated with icatibant and had sufficient icatibant plasma concentration-time measurements to derive primary PK parameters. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Time to peak concentration (Tmax) of a single SC dose of icatibant was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
n=10 Participants
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Peak Concentration (Tmax) of a Single Subcutaneous (SC) Dose of Icatibant
0.55 Hour (h)
Standard Deviation 0.19
0.42 Hour (h)
Standard Deviation 0.13
0.57 Hour (h)
Standard Deviation 0.17

PRIMARY outcome

Timeframe: Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Population: The PK population consisted of participants who were treated with icatibant and had sufficient icatibant plasma concentration-time measurements to derive primary PK parameters. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Maximum plasma concentration (Cmax) of a single SC dose of icatibant was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
n=10 Participants
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Maximum Plasma Concentration (Cmax) of a Single Subcutaneous (SC) Dose of Icatibant
805 Nanogram per milliliter (ng/mL)
Standard Deviation 125
659 Nanogram per milliliter (ng/mL)
Standard Deviation 158
761 Nanogram per milliliter (ng/mL)
Standard Deviation 133

PRIMARY outcome

Timeframe: Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Population: The PK population consisted of participants who were treated with icatibant and had sufficient icatibant plasma concentration time measurements to derive primary PK parameters. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Total plasma clearance (CL/F) of a single SC dose of icatibant was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=6 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
n=10 Participants
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Total Plasma Clearance (CL/F) of a Single Subcutaneous (SC) Dose of Icatibant
13.1 Milliliters per minute (mL/min)
Standard Deviation 3.42
10.8 Milliliters per minute (mL/min)
Standard Deviation 4.63
19.3 Milliliters per minute (mL/min)
Standard Deviation 4.84

PRIMARY outcome

Timeframe: Pre-dose; 0.25, 0.5, 0.75, 1, 2, and 4 hours post-dose on Day 1

Population: The PK population consisted of participants who were treated with icatibant and had sufficient icatibant plasma concentration time measurements to derive primary PK parameters. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Area under the plasma concentration-time curve from time zero to 4 hours post-dose (AUC0-4) of a single SC dose of icatibant was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
n=10 Participants
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Area Under the Plasma Concentration-time Curve From Time Zero to 4 Hours Post-dose (AUC0-4) of a Single Subcutaneous (SC) Dose of Icatibant
1448 Hour*nanogram per milliliter (h*ng/mL)
Standard Deviation 304
1241 Hour*nanogram per milliliter (h*ng/mL)
Standard Deviation 319
1335 Hour*nanogram per milliliter (h*ng/mL)
Standard Deviation 211

PRIMARY outcome

Timeframe: Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Population: The PK population consisted of participants who were treated with icatibant and had sufficient icatibant plasma concentration time measurements to derive primary PK parameters. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Area under the plasma concentration-time curve from time zero to 6 hours post-dose (AUC0-t) of a single SC dose of icatibant was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
n=10 Participants
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Area Under the Plasma Concentration-time Curve From Time Zero to 6 Hours Post-dose (AUC0-t) of a Single Subcutaneous (SC) Dose of Icatibant
1573 h*ng/mL
Standard Deviation 372
1289 h*ng/mL
Standard Deviation 325
1398 h*ng/mL
Standard Deviation 225

PRIMARY outcome

Timeframe: Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Population: The PK population consisted of participants who were treated with icatibant and had sufficient icatibant plasma concentration time measurements to derive primary PK parameters. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Area under the plasma concentration-time curve from time zero to infinity (AUC0-inf) of a single SC dose of icatibant was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=6 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
n=10 Participants
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Area Under the Plasma Concentration-time Curve From Time Zero to Infinity (AUC0-inf) of a Single Subcutaneous (SC) Dose of Icatibant
1710 h*ng/mL
Standard Deviation 569
1243 h*ng/mL
Standard Deviation 244
1416 h*ng/mL
Standard Deviation 229

PRIMARY outcome

Timeframe: Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Population: The PK population consisted of participants who were treated with icatibant and had sufficient icatibant plasma concentration time measurements to derive primary PK parameters. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Volume of distribution (Vz/F) of a single SC dose of icatibant was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=6 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
n=10 Participants
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Volume of Distribution (Vz/F) of a Single Subcutaneous (SC) Dose of Icatibant
23.5 Liters (L)
Standard Deviation 13.9
12.5 Liters (L)
Standard Deviation 5.28
25.4 Liters (L)
Standard Deviation 8.87

PRIMARY outcome

Timeframe: Pre-dose; 0.25, 0.5, 0.75, 1, 2, 4 and 6 hours post-dose on Day 1

Population: The PK population consisted of participants who were treated with icatibant and had sufficient icatibant plasma concentration time measurements to derive primary PK parameters. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Elimination half-life (t1/2) of a single SC dose of icatibant was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=6 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
n=10 Participants
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Elimination Half-life (t1/2) of a Single Subcutaneous (SC) Dose of Icatibant
1.34 h
Standard Deviation 0.96
0.80 h
Standard Deviation 0.04
0.90 h
Standard Deviation 0.10

PRIMARY outcome

Timeframe: Pre-dose up to 97 days post-dose

Population: Safety population consisted of participants who were treated with icatibant at least once during the study.

Vital signs included pulse rate, blood pressure, respiration rate, and temperature. The number of participants who reported clinically significant changes in vital signs were reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=21 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants With Clinically Significant Changes in Vital Signs
0 Participants
0 Participants

PRIMARY outcome

Timeframe: 6 - 8 hours post-dose on Day 1

Population: Safety population consisted of participants who were treated with icatibant at least once during the study.

A standard 12-lead ECG was performed after 10 minutes at rest when the participant was seated or supine following treatment. The number of participants who reported clinically significant changes in ECGs were reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=21 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants With Clinically Significant Changes in Electrocardiograms (ECGs)
0 Participants
0 Participants

PRIMARY outcome

Timeframe: Pre-dose up to 97 days post-dose

Population: Safety population consisted of participants who were treated with icatibant at least once during the study.

Clinical laboratory evaluations included clinical chemistry (including liver function tests), hematology, urinalysis. The number of participants who reported clinically significant changes in clinical laboratory evaluations were reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=21 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants With Clinically Significant Changes in Clinical Laboratory Evaluations
0 Participants
0 Participants

PRIMARY outcome

Timeframe: Pre-dose up to 97 days post-dose

Population: Safety population consisted of participants who were treated with icatibant at least once during the study.

The number of participants who reported anti-icatibant antibodies were reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=21 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants Who Reported Presence of Anti-icatibant Antibodies
0 Participants
0 Participants

PRIMARY outcome

Timeframe: From the start of study drug administration up to 97 days post-dose

Population: Safety population consisted of participants who were treated with icatibant at least once during the study.

An AE was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in a clinical study, whether or not considered investigational product related.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=21 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants With Adverse Events (AEs)
11 Participants
2 Participants

PRIMARY outcome

Timeframe: 1 h post-dose on Day 1 up to 9 days post-dose

Population: Safety population consisted of participants who were treated with icatibant at least once during the study. Here the number of participants analyzed signifies participants who were evaluable for this measure.

The number of participants with injection site reactions (erythema, swelling, burning sensation, itching/pruritus, warm sensation, cutaneous pain, or other) that occured after initial icatibant administration was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=21 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 1
Any Reaction
20 Participants
9 Participants
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 1
Any Severe Reaction
2 Participants
0 Participants

PRIMARY outcome

Timeframe: 1 h post-dose up to 9 days post-dose

Population: Safety population consisted of participants who were treated with icatibant at least once during the study. Here the number of participants analyzed signifies participants who received subsequent icatibant exposures 2 and 3.

The number of participants with injection site reactions (erythema, swelling, burning sensation, itching/pruritus, warm sensation, cutaneous pain, or other) that occurred after subsequent icatibant administration by study-site personnel (health care practitioner \[HCP\] administration) or by caregiver/self (caregiver administration) was reported. In the below table, E-2 refers to icatibant exposure 2 and E-3 refers to icatibant exposure 3.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 2 and 3
E-2: HCP Administration: Any Reaction
1 Participants
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 2 and 3
E-2: HCP Administration: Any Severe Reaction
0 Participants
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 2 and 3
E-2: Caregiver Administration: Any Reaction
8 Participants
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 2 and 3
E-2: Caregiver Administration: Any Severe Reaction
3 Participants
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 2 and 3
E-3: HCP Administration: Any Reaction
1 Participants
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 2 and 3
E-3: HCP Administration: Any Severe Reaction
0 Participants
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 2 and 3
E-3: Caregiver Administration: Any Reaction
7 Participants
Number of Participants Who Reported Injection Site Reactions (ISR) for Icatibant Exposure Number 2 and 3
E-3: Caregiver Administration: Any Severe Reaction
1 Participants

PRIMARY outcome

Timeframe: Pre-dose up to 97 days post-dose

Population: Safety population consisted of participants who were treated with icatibant at least once during the study.

Reproductive hormone levels of follicle-stimulating hormone (FSH), luteinizing hormone (LH), estradiol, and progesterone in females, and FSH, LH, and testosterone in males were measured. The number of participants with clinically significant changes in reproductive hormones was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=21 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants With Clinically Significant Changes in Reproductive Hormones
0 Participants
0 Participants

SECONDARY outcome

Timeframe: From start of study drug administration up to 8.5 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants who were evaluable for this measure.

The TOSR was defined as the duration of time in hours from study drug administration to the earliest time post-treatment at which there was at least a 20 percent (%) improvement in the average post-treatment symptom score with no worsening of any single component score for the initial icatibant exposure. The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of hereditary angioedema (HAE) using the following 5-point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). TOSR for participants who received initial icatibant administration was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 1
1.0 h
Interval 1.0 to 2.0
1.0 h
Interval 1.0 to 2.0

SECONDARY outcome

Timeframe: From start of study drug administration up to 12 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants who received subsequent icatibant exposures 2 and 3.

The TOSR was defined as the duration of time in hours from study drug administration to the earliest time post-treatment at which there was at least a 20% improvement in the composite (or average) post-treatment symptom score with no worsening of any single component score. The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of HAE using the following 5-point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). TOSR for participants who received subsequent icatibant administration by HCP administration or by caregiver/ self-administration was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-2: HCP Administration
4.0 h
Percentage confidence interval (%CI) was not calculated due to less number of participants.
Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-2: Caregiver Administration
1.0 h
Interval 1.0 to 2.3
Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-3: HCP Administration
1.0 h
%CI was not calculated due to less number of participants.
Time to Onset of Symptom Relief (TOSR) for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-3:Caregiver Administration
1.1 h
Interval 1.0 to 3.0

SECONDARY outcome

Timeframe: From start of study drug administration up to 52 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants with FPS-R data.

The TOSR was defined as the earliest time at which the post-treatment score improved by at least one level. Participants of 4 years age and older self-assessed their HAE-related pain using the FPS-R instrument. FPS-R is a self-reported measure used to assess the intensity of children's pain and it is scored using a 0 to 10 scale (0=no pain to 10=very much pain). TOSR for participants who received initial icatibant administration was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=9 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=6 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 1
1.0 h
Interval 0.6 to 1.0
0.9 h
Interval 0.8 to 1.0

SECONDARY outcome

Timeframe: From start of study drug administration up to 28 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants with FPS-R data.

The TOSR was defined as the earliest time at which the post-treatment score improved by at least one level. Participants of 4 years age and older self-assessed their HAE-related pain using the FPS-R instrument. FPS-R is a self-reported measure used to assess the intensity of children's pain and it is scored using a 0 to 10 scale (0=no pain to 10=very much pain). TOSR for participants who received subsequent icatibant administration by HCP administration or by caregiver/ self-administration was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-2: HCP Administration
3.0 h
%CI was not calculated due to less number of participants.
Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-2: Caregiver Administration
1.0 h
Interval 1.0 to 1.2
Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-3: HCP Administration
1.0 h
%CI was not calculated due to less number of participants.
Time to Onset of Symptom Relief (TOSR) for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-3:Caregiver Administration
1.1 h
Interval 1.0 to 1.2

SECONDARY outcome

Timeframe: From start of study drug administration up to 8.5 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants of 4 years and younger with FLACC data.

The TOSR was defined as the earliest time at which a 20% improvement was seen in the total post-treatment score. Participants of 4 years age and younger underwent investigator assessment of HAE-related pain (cutaneous, abdominal, and laryngeal) using the FLACC comportmental pain scale. Each of the 5 categories was scored from 0 to 2. Face(F): 0 (no particular expression/smile) - 2 (frequent to constant frown clenched jaw quivering chin); Legs(L): 0 (normal position/relaxed) - 2 (kicking/legs drawn up); Activity(A): 0 (lying quietly, normal position, moves easily) - 2 (arched rigid/jerking); Cry(C): 0 (No cry \[awake/asleep\]) - 2 (crying steadily/screams/sobs or frequent complaints); Consolability(C): 0 (content/relaxed) - 2 (difficult to console/comfort), resulting in a total score between 0 and 10.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=1 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Onset of Symptom Relief (TOSR) for Faces, Legs, Activity, Cry, and Consolability (FLACC) Scores
1.0 h
%CI was not calculated due to less number of participants.

SECONDARY outcome

Timeframe: From start of study drug administration up to 8.5 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Time to minimum symptom was defined as the duration of time in hours from study drug administration to the earliest time post-treatment at which all symptoms were either mild or absent for the investigator-reported symptom score. The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of HAE using the following 5-point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). Time to minimum symptom for participants who received initial icatibant administration was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=10 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Minimum Symptoms for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 1
1.0 h
Interval 1.0 to 2.0
1.9 h
Interval 1.0 to 2.0

SECONDARY outcome

Timeframe: From start of study drug administration up to 12 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Time to minimum symptom was defined as the duration of time in hours from study drug administration to the earliest time post-treatment at which all symptoms were either mild or absent for the investigator-reported symptom score. The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of HAE using the following 5-point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). Time to minimum symptom for participants who received subsequent icatibant administration by HCP administration or by caregiver/ self-administration was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Minimum Symptoms for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-2: Caregiver Administration
1.2 h
Interval 1.0 to 2.0
Time to Minimum Symptoms for Composite Investigator-Assessed Symptom Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-3: Caregiver Administration
2.2 h
Interval 1.0 to
The data was not calculated due to analysis method limitation.

SECONDARY outcome

Timeframe: From start of study drug administration up to 52 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants with FPS-R data.

Time to minimum symptoms was defined as the duration of time in hours from study drug administration to the earliest time at which post-treatment score improved to zero (or no pain). Participants of 4 years of age and older self-assessed their HAE-related pain using the FPS-R instrument. FPS-R is a self-reported measure used to assess the intensity of children's pain and it is scored using a 0 to 10 scale (0=no pain to 10=very much pain). Time to minimum symptom for participants who received initial icatibant administration was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=6 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Minimum Symptom for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 1
3.8 h
Interval 1.0 to 6.8
2.4 h
Interval 1.9 to 5.3

SECONDARY outcome

Timeframe: From start of study drug administration up to 28 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants with FPS-R data.

Time to minimum symptoms was defined as the duration of time in hours from study drug administration to the earliest time at which post-treatment score improved to zero (or no pain). Participants of 4 years of age and older self-assessed their HAE-related pain using the FPS-R instrument. FPS-R is a self-reported measure used to assess the intensity of children's pain and it is scored using a 0 to 10 scale (0=no pain to 10=very much pain). Time to minimum symptom for participants who received subsequent icatibant administration by HCP administration or by caregiver/ self-administration was reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Minimum Symptom for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-2: HCP Administration
3.0 h
The %CI was not calculated due to less number of participants.
Time to Minimum Symptom for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-2: Caregiver Administration
2.1 h
Interval 1.0 to 4.0
Time to Minimum Symptom for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-3: HCP Administration
5.8 h
The %CI was not calculated due to less number of participants.
Time to Minimum Symptom for Faces Pain Scale-Revised (FPS-R) Scores for Icatibant Exposure Number 2 and 3
Icatibant Exposure-3:Caregiver Administration
24.0 h
Interval 3.8 to 24.2

SECONDARY outcome

Timeframe: From start of study drug administration up to 8.5 hours post-dose

Population: Participants from efficacy population with FLACC data.

Time to minimum symptoms was defined as the duration of time in hours from study drug administration to the earliest time at which the total post-treatment score improved to zero. Participants of 4 years age and younger underwent investigator assessment of HAE-related pain (cutaneous, abdominal, and laryngeal) using the FLACC comportmental pain scale. Each of the 5 categories was scored from 0 to 2. (F) Face: 0 (no particular expression/smile) - 2 (frequent to constant frown clenched jaw quivering chin); (L) Legs: 0 (normal position/relaxed) - 2 (kicking/legs drawn up); (A) Activity: 0 (lying quietly, normal position, moves easily) - 2 (arched rigid/jerking); (C) Cry: 0 (No cry \[awake/asleep\]) - 2 (crying steadily/screams/sobs or frequent complaints); (C) Consolability: 0 (content/relaxed) - 2 (difficult to console/comfort), resulting in a total score between 0 and 10.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=1 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Minimum Symptom for Faces, Legs, Activity, Cry, and Consolability (FLACC) Scores
1.0 h
The %CI was not calculated due to less number of participants.

SECONDARY outcome

Timeframe: From the start of study drug administration up to 52 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants who were evaluable for this measure.

Rescue medication was any medication used after the administration of icatibant which, in the opinion of the investigator, was immediately necessary to alleviate acute symptoms which are judged by the investigator as resultant from the current HAE attack. Time to first use of rescue medication prior to the onset of symptom relief was calculated from the time of study drug administration to the first use of rescue medication prior to the onset of symptom relief. This analysis was not performed since as per protocol, "This analysis will only be performed if there are at least 5 participants for a given attack who used rescue medication prior to attaining symptom relief".

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Time to Use of Rescue Medication for the Treatment of Symptoms of the Hereditary Angioedema (HAE) Attack Following Study Drug Administration
NA h
This analysis was planned to be performed if there were at least 5 participants for a given attack who used rescue medication prior to attaining symptom relief.
NA h
This analysis was planned to be performed if there were at least 5 participants for a given attack who used rescue medication prior to attaining symptom relief.

SECONDARY outcome

Timeframe: From 2 hours post-dose to 4 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. Here the number of participants analyzed signifies participants who evaluable for this endpoint.

The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of HAE using the following 5- point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). The number of participants with a worsened severity of HAE symptoms at 4 hours post-dose from 2 hours postdose were reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
n=11 Participants
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=11 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 1
Abdominal Tenderness
1 Participants
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 1
Nausea
0 Participants
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 1
Vomiting
0 Participants
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 1
Diarrhea
0 Participants
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 1
Skin Pain
0 Participants
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 1
Erythema
0 Participants
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 1
Skin Irritation
0 Participants
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 1
Skin Swelling
0 Participants
0 Participants

SECONDARY outcome

Timeframe: From 2 hours post-dose to 4 hours post-dose

Population: Efficacy population consisted of participants who were treated with icatibant for their first and any additional attacks during the study. The number of participants analyzed signifies participants evaluable for this endpoint.

The investigator used a symptom score to assess the severities of symptoms of acute cutaneous, abdominal, and laryngeal attacks of HAE using the following 5- point scale: 0=none (absence of symptoms), 1=mild (no to mild interference with daily activities), 2=moderate (moderate interference with daily activities), 3=severe (severe interference with daily activities) and 4=very severe (very severe interference with daily activities). The number of participants with a worsened severity of HAE symptoms at 4 hours post-dose from 2 hours post-dose were reported.

Outcome measures

Outcome measures
Measure
Pubertal/Postpubertal
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Prepubertal
n=9 Participants
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Postpubertal: Without Acute Attack
Participants without acute attack received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Abdominal Tenderness
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2:CA: Abdominal Tenderness
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Nausea
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Nausea
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2:HCPA: Vomiting
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Vomiting
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Diarrhea
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Diarrhea
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Skin Pain
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Skin Pain
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Erythema
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Erythema
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Skin Irritation
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Skin Irritation
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Skin Swelling
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Skin Swelling
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Dysphagia
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Dysphagia
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Voice Change
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Voice Change
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Breathing Difficulties
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Breathing Difficulties
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Stridor
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: CA: Stridor
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2: HCPA: Asphyxia
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-2:CA: Asphyxia
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Abdominal Tenderness
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Abdominal Tenderness
1 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Nausea
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Nausea
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Vomiting
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Vomiting
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Diarrhea
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Diarrhea
1 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Skin Pain
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Skin Pain
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Erythema
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Erythema
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Skin Irritation
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Skin Irritation
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Skin Swelling
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Skin Swelling
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Dysphagia
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Dysphagia
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Voice Change
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Voice Change
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Breathing Difficulties
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Breathing Difficulties
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Stridor
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Stridor
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: HCPA: Asphyxia
0 Participants
Number of Participants With Worsened Intensity of Clinical Hereditary Angioedema (HAE) Symptoms Between 2 and 4 Hours After Treatment With Icatibant Exposure Number 2 and 3
E-3: CA: Asphyxia
0 Participants

Adverse Events

Prepubertal

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Pubertal/Post-pubertal

Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths

Overall

Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Prepubertal
n=11 participants at risk
Participants received a single SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region.
Pubertal/Post-pubertal
n=21 participants at risk
Participants received a SC injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region and participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Overall
n=32 participants at risk
Participants received a single subcutaneous(SC) injection of 0.4 mg/kg icatibant (up to a maximal dose of 30 mg) in the abdominal region. Pubertal/postpubertal participants after receiving initial treatment with icatibant, who subsequently experienced an acute hereditary angioedema (HAE) attack continued to receive treatment with icatibant as a single SC administration per attack for a total of 3 eligible icatibant exposures.
Blood and lymphatic system disorders
Anaemia
9.1%
1/11 • Number of events 1 • From start of study drug administration up to 187 days
0.00%
0/21 • From start of study drug administration up to 187 days
3.1%
1/32 • Number of events 1 • From start of study drug administration up to 187 days
Musculoskeletal and connective tissue disorders
Arthralgia
9.1%
1/11 • Number of events 1 • From start of study drug administration up to 187 days
0.00%
0/21 • From start of study drug administration up to 187 days
3.1%
1/32 • Number of events 1 • From start of study drug administration up to 187 days
Eye disorders
Conjunctivitis
0.00%
0/11 • From start of study drug administration up to 187 days
9.5%
2/21 • Number of events 5 • From start of study drug administration up to 187 days
6.2%
2/32 • Number of events 5 • From start of study drug administration up to 187 days
Eye disorders
Conjunctivitis allergic
0.00%
0/11 • From start of study drug administration up to 187 days
9.5%
2/21 • Number of events 9 • From start of study drug administration up to 187 days
6.2%
2/32 • Number of events 9 • From start of study drug administration up to 187 days
Gastrointestinal disorders
Toothache
0.00%
0/11 • From start of study drug administration up to 187 days
9.5%
2/21 • Number of events 2 • From start of study drug administration up to 187 days
6.2%
2/32 • Number of events 2 • From start of study drug administration up to 187 days
General disorders
Pyrexia
0.00%
0/11 • From start of study drug administration up to 187 days
9.5%
2/21 • Number of events 2 • From start of study drug administration up to 187 days
6.2%
2/32 • Number of events 2 • From start of study drug administration up to 187 days
Infections and infestations
Nasopharyngitis
0.00%
0/11 • From start of study drug administration up to 187 days
14.3%
3/21 • Number of events 4 • From start of study drug administration up to 187 days
9.4%
3/32 • Number of events 4 • From start of study drug administration up to 187 days
Infections and infestations
Upper respiratory tract infection
9.1%
1/11 • Number of events 1 • From start of study drug administration up to 187 days
9.5%
2/21 • Number of events 3 • From start of study drug administration up to 187 days
9.4%
3/32 • Number of events 4 • From start of study drug administration up to 187 days
Injury, poisoning and procedural complications
Epiphyseal fracture
9.1%
1/11 • Number of events 1 • From start of study drug administration up to 187 days
0.00%
0/21 • From start of study drug administration up to 187 days
3.1%
1/32 • Number of events 1 • From start of study drug administration up to 187 days
Injury, poisoning and procedural complications
Thermal burn
9.1%
1/11 • Number of events 1 • From start of study drug administration up to 187 days
0.00%
0/21 • From start of study drug administration up to 187 days
3.1%
1/32 • Number of events 1 • From start of study drug administration up to 187 days
Investigations
Nitrite urine present
9.1%
1/11 • Number of events 1 • From start of study drug administration up to 187 days
0.00%
0/21 • From start of study drug administration up to 187 days
3.1%
1/32 • Number of events 1 • From start of study drug administration up to 187 days
Metabolism and nutrition disorders
Dehydration
9.1%
1/11 • Number of events 1 • From start of study drug administration up to 187 days
0.00%
0/21 • From start of study drug administration up to 187 days
3.1%
1/32 • Number of events 1 • From start of study drug administration up to 187 days
Nervous system disorders
Headache
9.1%
1/11 • Number of events 2 • From start of study drug administration up to 187 days
4.8%
1/21 • Number of events 1 • From start of study drug administration up to 187 days
6.2%
2/32 • Number of events 3 • From start of study drug administration up to 187 days
Respiratory, thoracic and mediastinal disorders
Bronchospasm
9.1%
1/11 • Number of events 2 • From start of study drug administration up to 187 days
0.00%
0/21 • From start of study drug administration up to 187 days
3.1%
1/32 • Number of events 2 • From start of study drug administration up to 187 days
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
0.00%
0/11 • From start of study drug administration up to 187 days
9.5%
2/21 • Number of events 2 • From start of study drug administration up to 187 days
6.2%
2/32 • Number of events 2 • From start of study drug administration up to 187 days

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Results disclosure agreements

  • Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
  • Publication restrictions are in place

Restriction type: OTHER