Trial Outcomes & Findings for EASSI - Evaluation of the Safety of Self-Administration With Icatibant (NCT NCT00997204)
NCT ID: NCT00997204
Last Updated: 2021-06-11
Results Overview
Clinical safety of self-treatment of acute HAE attacks with s.c. injections of icatibant was assessed by calculating the number of AEs occurred during the study. Only those adverse events occurring up to the earlier of 7 days from the start of the naive phase, study discontinuation and start of the self-administration phase are assessed. The Local Tolerability Assessment tool was used. Subjects and Investigators graded erythema/reddening, swelling, burning, pruritus/itching, warm sensation, and skin pain on a 0 to 3 severity scale.
COMPLETED
PHASE3
151 participants
7 days from the beginning of each phase
2021-06-11
Participant Flow
Patients were screened for entry based on their known medical histories (HAE attacks) and previous exposure to a treatment (naïve or not). 151 were enrolled and trained in the self-administration. 47 of these subjects did not have an acute attack of HAE treated with icatibant during this study and were included in the untreated population.
Participant milestones
| Measure |
Naive Subjects/ Naive Treatment Phase
Patients who had never received icatibant before this phase, got treatment of Acute HAE Attack with SC icatibant (30 mg)Administered at Site by Health Care Provider.
|
Non-Naive Subjects/ Self-administration Phase
Subjects who had received treatment for HAE with icatibant in previous clinical trials or had been previously treated with the marketed product Firazyr®, got Treatment of Acute HAE Attack with SC icatibant (30 mg)Self-Administered.
|
|---|---|---|
|
Naive Treatment Phase
STARTED
|
25
|
79
|
|
Naive Treatment Phase
COMPLETED
|
19
|
0
|
|
Naive Treatment Phase
NOT COMPLETED
|
6
|
79
|
|
Self-administration Phase
STARTED
|
19
|
78
|
|
Self-administration Phase
COMPLETED
|
19
|
77
|
|
Self-administration Phase
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
EASSI - Evaluation of the Safety of Self-Administration With Icatibant
Baseline characteristics by cohort
| Measure |
Non-Naive Patients
n=79 Participants
Patients who previously treated with icatibant in clinical studies or with commercial Firazyr® and got the treatment during the self-administered phase
|
Naive Patients
n=25 Participants
Patients who had never received icatibant and treated in both the Naive treatment phase and the Self-administered phase
|
Total
n=104 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
40.607 years
STANDARD_DEVIATION 13.044 • n=99 Participants
|
44.68 years
STANDARD_DEVIATION 16.53 • n=107 Participants
|
41.58 years
STANDARD_DEVIATION 13.98 • n=206 Participants
|
|
Sex: Female, Male
Female
|
52 Participants
n=99 Participants
|
16 Participants
n=107 Participants
|
68 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
27 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
36 Participants
n=206 Participants
|
|
Region of Enrollment
France
|
6 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Region of Enrollment
Argentina
|
18 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
23 Participants
n=206 Participants
|
|
Region of Enrollment
Spain
|
9 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
11 Participants
n=206 Participants
|
|
Region of Enrollment
Denmark
|
5 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Region of Enrollment
Austria
|
9 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
14 Participants
n=206 Participants
|
|
Region of Enrollment
Israel
|
15 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
|
Region of Enrollment
Germany
|
9 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
14 Participants
n=206 Participants
|
|
Region of Enrollment
Italy
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Region of Enrollment
Switzerland
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Region of Enrollment
United Kingdom
|
6 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: 7 days from the beginning of each phaseClinical safety of self-treatment of acute HAE attacks with s.c. injections of icatibant was assessed by calculating the number of AEs occurred during the study. Only those adverse events occurring up to the earlier of 7 days from the start of the naive phase, study discontinuation and start of the self-administration phase are assessed. The Local Tolerability Assessment tool was used. Subjects and Investigators graded erythema/reddening, swelling, burning, pruritus/itching, warm sensation, and skin pain on a 0 to 3 severity scale.
Outcome measures
| Measure |
Naive Subjects Administered Icatibant by Health Care Provider
n=22 Participants
The first HAE attack of naïve subjects enrolled in the study was treated at the study site, where a Health Care Provider administered icatibant to the subject. 3 subjects (of the original 25 enrolled in the naive treatment phase)self-administered icatibant while observed bu HCP, as opposed to having the HCP perform the injection. these data were not included in the naive treatment safety analyses.
|
Subjects Who Self-administered Icatibant (Naive)
n=19 Participants
Naive subjects self-administered the study drug at home or other site convenient to the subject, but not at the investigational site, nor under HCP-supervision.
|
Subjects Who Self-administered Icatibant (Non-naive)
n=78 Participants
Non-Naive subjects self-administered the study drug at home or other site convenient to the subject, but not at the investigational site, nor under HCP-supervision.
|
|---|---|---|---|
|
Number of Participants With Adverse Events in Self-treatment of Acute HAE Attacks With s.c. Injections of Icatibant
|
11 participants
|
6 participants
|
27 participants
|
SECONDARY outcome
Timeframe: 48 hours post-doseSubjects assessed angioedema attack symptoms using the visual analogue scale (VAS) for skin pain, skin swelling and abdominal pain. The VAS is a continuous scale comprised of a 100 mm in length line, anchored by 2 verbal descriptors, one for each symptom extreme 0 (no pain) and 100 (worst pain). The respondent is asked to place a mark on the VAS line (any where between 0 and 100 mm) at the point that represents their pain intensity. The score is determined by measuring the distance (mm) on the line between the "no pain" anchor and the patient's mark, providing a range of scores from 0-100. A higher score indicates greater pain intensity. Score interpretation is: no pain (0-4 mm), mild pain (5-44 mm), moderate pain (45-74 mm), and severe pain (75-100 mm). Symptom relief is defined as at least a 50% reduction in a pre-dose VAS score of 30 mm or greater. The time to onset of symptom relief is defined as the first of 3 consecutive assessments at which symptom relief was observed.
Outcome measures
| Measure |
Naive Subjects Administered Icatibant by Health Care Provider
n=22 Participants
The first HAE attack of naïve subjects enrolled in the study was treated at the study site, where a Health Care Provider administered icatibant to the subject. 3 subjects (of the original 25 enrolled in the naive treatment phase)self-administered icatibant while observed bu HCP, as opposed to having the HCP perform the injection. these data were not included in the naive treatment safety analyses.
|
Subjects Who Self-administered Icatibant (Naive)
n=19 Participants
Naive subjects self-administered the study drug at home or other site convenient to the subject, but not at the investigational site, nor under HCP-supervision.
|
Subjects Who Self-administered Icatibant (Non-naive)
n=78 Participants
Non-Naive subjects self-administered the study drug at home or other site convenient to the subject, but not at the investigational site, nor under HCP-supervision.
|
|---|---|---|---|
|
Clinical Efficacy of Self-treatment of Acute HAE Attacks With s.c. Injections of Icatibant, Time to Symptom Relief Using VAS Score for a Single Primary Symptom by Patient Cohort
|
2.0 Hours
Interval 1.0 to 4.2
|
3.1 Hours
Interval 2.0 to 4.0
|
2.0 Hours
Interval 1.0 to 5.3
|
Adverse Events
Naive Subjects Administered Icatibant by Health Care Provider
Subjects Who Self-administered Icatibant (Naive)
Subjects Who Self-administered Icatibant (Non-naive)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Naive Subjects Administered Icatibant by Health Care Provider
n=22 participants at risk
The first HAE attack of naïve subjects enrolled in the study was treated at the study site, where a Health Care Provider administered icatibant to the subject.
|
Subjects Who Self-administered Icatibant (Naive)
n=19 participants at risk
Naive subjects self-administered the study drug at home or other site convenient to the subject, but not at the investigational site, nor under HCP-supervision.
|
Subjects Who Self-administered Icatibant (Non-naive)
n=78 participants at risk
Non-naive subjects self-administered the study drug at home or other site convenient to the subject, but not at the investigational site, nor under HCP-supervision.
|
|---|---|---|---|
|
Congenital, familial and genetic disorders
Hereditary Angioedema
|
27.3%
6/22 • 7 days from the beginning of each phase
|
15.8%
3/19 • 7 days from the beginning of each phase
|
24.4%
19/78 • 7 days from the beginning of each phase
|
|
Nervous system disorders
Headache
|
4.5%
1/22 • 7 days from the beginning of each phase
|
5.3%
1/19 • 7 days from the beginning of each phase
|
2.6%
2/78 • 7 days from the beginning of each phase
|
|
Investigations
Blood pressure increased
|
0.00%
0/22 • 7 days from the beginning of each phase
|
5.3%
1/19 • 7 days from the beginning of each phase
|
0.00%
0/78 • 7 days from the beginning of each phase
|
|
Respiratory, thoracic and mediastinal disorders
Pharyngeal erythema
|
0.00%
0/22 • 7 days from the beginning of each phase
|
5.3%
1/19 • 7 days from the beginning of each phase
|
0.00%
0/78 • 7 days from the beginning of each phase
|
|
General disorders
Feeling Hot
|
0.00%
0/22 • 7 days from the beginning of each phase
|
5.3%
1/19 • 7 days from the beginning of each phase
|
0.00%
0/78 • 7 days from the beginning of each phase
|
|
General disorders
Local Swelling
|
0.00%
0/22 • 7 days from the beginning of each phase
|
5.3%
1/19 • 7 days from the beginning of each phase
|
0.00%
0/78 • 7 days from the beginning of each phase
|
|
General disorders
Edema Peripheral
|
0.00%
0/22 • 7 days from the beginning of each phase
|
5.3%
1/19 • 7 days from the beginning of each phase
|
0.00%
0/78 • 7 days from the beginning of each phase
|
|
General disorders
Localized edema
|
0.00%
0/22 • 7 days from the beginning of each phase
|
5.3%
1/19 • 7 days from the beginning of each phase
|
0.00%
0/78 • 7 days from the beginning of each phase
|
|
Skin and subcutaneous tissue disorders
Skin Lesion
|
0.00%
0/22 • 7 days from the beginning of each phase
|
5.3%
1/19 • 7 days from the beginning of each phase
|
0.00%
0/78 • 7 days from the beginning of each phase
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal edema
|
0.00%
0/22 • 7 days from the beginning of each phase
|
5.3%
1/19 • 7 days from the beginning of each phase
|
0.00%
0/78 • 7 days from the beginning of each phase
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60