Trial Outcomes & Findings for Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema (NCT NCT02052141)
NCT ID: NCT02052141
Last Updated: 2021-06-03
Results Overview
Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.
COMPLETED
PHASE3
12 participants
From start of treatment up to 12 weeks during each treatment period
2021-06-03
Participant Flow
The study was conducted in 10 study centers in the United States, European Union, Mexico, and Israel between 20 March 2014 (first participant first visit) and 04 May 2017 (last participant last visit).
A total of 16 participants were screened and of them, 12 were enrolled into the baseline observational period (12 weeks) and were randomized to receive the treatment in sequence A-B and B-A during this crossover study without a washout period.
Participant milestones
| Measure |
Treatment A-B (500 U/1000 U CINRYZE)
Participants received 500 units (U) of CINRYZE intravenous (IV) injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment A) during intervention period 1 followed by 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment B) during intervention period 2. There was no washout period between the two intervention periods.
|
Treatment B-A (1000 U/500 U CINRYZE)
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment B) during intervention period 1 followed by 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment A) during intervention period 2. There was no washout period between the two intervention periods.
|
|---|---|---|
|
Intervention Period 1
STARTED
|
5
|
7
|
|
Intervention Period 1
COMPLETED
|
5
|
7
|
|
Intervention Period 1
NOT COMPLETED
|
0
|
0
|
|
Intervention Period 2
STARTED
|
5
|
7
|
|
Intervention Period 2
COMPLETED
|
5
|
7
|
|
Intervention Period 2
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema
Baseline characteristics by cohort
| Measure |
Treatment A-B (500 U/1000 U CINRYZE)
n=5 Participants
Participants received 500 units (U) of CINRYZE intravenous (IV) injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment A) in Intervention period 1 followed by 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment B) in Intervention period 2. There was no washout period between two intervention periods.
|
Treatment B-A (1000 U/500 U CINRYZE)
n=7 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment B) in Intervention period 1 followed by 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment A) in Intervention period 2. There was no washout period between two intervention periods.
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
10.2 Years
STANDARD_DEVIATION 0.84 • n=5 Participants
|
9.4 Years
STANDARD_DEVIATION 1.51 • n=7 Participants
|
9.8 Years
STANDARD_DEVIATION 1.29 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From start of treatment up to 12 weeks during each treatment periodPopulation: Full Analysis Set (FAS) included all participants in the safety set who had at least 1 post-baseline primary efficacy assessment.
Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.
Outcome measures
| Measure |
Treatment B (1000 U CINRYZE)
n=12 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment A (500 U CINRYZE)
n=12 Participants
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
Normalized Number of Angioedema Attacks Per Month in a Treatment Period
|
0.7 Angioedema attacks per month
Standard Deviation 1.35
|
1.2 Angioedema attacks per month
Standard Deviation 1.53
|
SECONDARY outcome
Timeframe: From start of treatment up to 12 weeks during each treatment periodPopulation: FAS included all participants in the safety set who had at least 1 post-baseline primary efficacy assessment.
Severity of the angioedema attack sign/symptom was characterized as None: no symptom; Mild: noticeable symptom but easily tolerated by the participant and did not interfere with routine activities; Moderate: symptom interfered with the participant's ability to attend school or participate in family life and social/recreational activities; Severe: symptom significantly limited the participant's ability to attend school or participate in family life and social/recreational activities. Symptom severity score was assigned as Mild = 1, Moderate = 2 and Severe = 3. Cumulative attack severity score was the sum of the maximum symptom severity scores recorded for each angioedema attack in a treatment period. Cumulative attack-severity score normalized per month \[(raw score/number of days of participation in that treatment period)\*30.4\] was reported here. Cumulative attack-severity score normalized per month ranged from 0 to 10.4 and higher scores represent worse symptoms.
Outcome measures
| Measure |
Treatment B (1000 U CINRYZE)
n=12 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment A (500 U CINRYZE)
n=12 Participants
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
Cumulative Attack-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period
|
1.4 Score on a scale
Standard Deviation 2.68
|
2.0 Score on a scale
Standard Deviation 2.91
|
SECONDARY outcome
Timeframe: From start of treatment up to 12 weeks during each intervention periodPopulation: FAS included all participants in the safety set who had at least 1 post-baseline primary efficacy assessment.
Severity of the angioedema attack sign/symptom was characterized as None: no symptom; Mild: noticeable but easily tolerated by the participant and did not interfere with routine activities; Moderate: interfered with the participant's ability to attend school or participate in family life and social/recreational activities; Severe: significantly limited the participant's ability to attend school or participate in family life and social/recreational activities. Symptom severity score was assigned as Mild = 1, Moderate = 2 and Severe = 3. Cumulative daily-severity score was the sum of the severity scores recorded for every day of reported symptoms in a treatment period. Cumulative daily-severity score normalized per month \[(raw score/number of days of participation in that treatment period)\*30.4\] was reported here. Cumulative daily-severity score normalized per month ranged from 0 to 15.6 and higher scores represent worse symptoms.
Outcome measures
| Measure |
Treatment B (1000 U CINRYZE)
n=12 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment A (500 U CINRYZE)
n=12 Participants
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
Cumulative Daily-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period
|
2.2 Score on a scale
Standard Deviation 3.50
|
4.1 Score on a scale
Standard Deviation 5.01
|
SECONDARY outcome
Timeframe: From start of treatment up to 12 weeks during each intervention periodPopulation: FAS included all participants in the safety set who had at least 1 post-baseline primary efficacy assessment.
Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks requiring acute treatment was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.
Outcome measures
| Measure |
Treatment B (1000 U CINRYZE)
n=12 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment A (500 U CINRYZE)
n=12 Participants
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
Normalized Number of Angioedema Attacks Per Month Requiring Acute Treatment in a Treatment Period
|
0.4 Angioedema attacks per month
Standard Deviation 1.27
|
0.7 Angioedema attacks per month
Standard Deviation 1.5
|
SECONDARY outcome
Timeframe: From start of study treatment up to 25 weeksPopulation: Safety set included all participants who received at least 1 dose of investigational product.
An adverse event (AE) was any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurring in a participant participating in a clinical study with the sponsor's product, regardless of causal relationship. TEAEs were defined as events that started or worsened on or after the date and time of the first dose of investigational product and up to 7 days after the last dose of investigational product.
Outcome measures
| Measure |
Treatment B (1000 U CINRYZE)
n=12 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment A (500 U CINRYZE)
n=12 Participants
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Dose Group
|
11 Participants
|
10 Participants
|
SECONDARY outcome
Timeframe: Pre-dose and 1 hour (h) post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention periodPopulation: Pharmacokinetic (PK) set consisted of all pariticipants in the safety set with no major deviations related to investigational product intake and evaluable PK profiles.
C1 INH antigen concentration in plasma was determined using an automated nephelometric assay.
Outcome measures
| Measure |
Treatment B (1000 U CINRYZE)
n=12 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment A (500 U CINRYZE)
n=12 Participants
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen
Week 1 (Pre-dose 1)
|
0.0736 Gram per liter (g/L)
Standard Deviation 0.02885
|
0.0945 Gram per liter (g/L)
Standard Deviation 0.03294
|
|
Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen
Week 1 (1 h post-dose 1)
|
0.2084 Gram per liter (g/L)
Standard Deviation 0.08757
|
0.1819 Gram per liter (g/L)
Standard Deviation 0.04331
|
|
Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen
Week 6 (Pre-dose 12)
|
0.1068 Gram per liter (g/L)
Standard Deviation 0.03098
|
0.0965 Gram per liter (g/L)
Standard Deviation 0.03129
|
|
Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen
Week 6 (1 h post-dose 12)
|
0.2543 Gram per liter (g/L)
Standard Deviation 0.05499
|
0.1631 Gram per liter (g/L)
Standard Deviation 0.04188
|
|
Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen
Week 12 (Pre-dose 24)
|
0.1002 Gram per liter (g/L)
Standard Deviation 0.04420
|
0.0823 Gram per liter (g/L)
Standard Deviation 0.02758
|
|
Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen
Week 12 (1 h post-dose 24)
|
0.2396 Gram per liter (g/L)
Standard Deviation 0.04511
|
0.1621 Gram per liter (g/L)
Standard Deviation 0.02990
|
|
Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen
Week 12 (2 h post-dose 24)
|
0.2070 Gram per liter (g/L)
Standard Deviation 0.01838
|
0.1440 Gram per liter (g/L)
Standard Deviation 0.00400
|
|
Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen
Week 12 (4 h post-dose 24)
|
0.1770 Gram per liter (g/L)
Standard Deviation 0.02970
|
0.1440 Gram per liter (g/L)
Standard Deviation 0.01131
|
|
Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen
Week 12 (8 h post-dose 24)
|
0.1790 Gram per liter (g/L)
Standard Deviation 0.03100
|
0.1280 Gram per liter (g/L)
Standard Deviation 0.00990
|
SECONDARY outcome
Timeframe: Pre-dose and 1 h post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention periodPopulation: PK set consisted of all pariticipants in the safety set with no major deviations related to investigational product intake and evaluable PK profiles.
The functional activity of C1 INH in plasma samples was determined by a chromogenic assay.
Outcome measures
| Measure |
Treatment B (1000 U CINRYZE)
n=12 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment A (500 U CINRYZE)
n=12 Participants
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma
Week 1 (Pre-dose 1)
|
0.210 Units per milliliter (U/mL)
Standard Deviation 0.1282
|
0.290 Units per milliliter (U/mL)
Standard Deviation 0.0914
|
|
C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma
Week 1 (1 h post-dose 1)
|
0.725 Units per milliliter (U/mL)
Standard Deviation 0.3100
|
0.575 Units per milliliter (U/mL)
Standard Deviation 0.1358
|
|
C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma
Week 6 (Pre-dose 12)
|
0.336 Units per milliliter (U/mL)
Standard Deviation 0.0933
|
0.297 Units per milliliter (U/mL)
Standard Deviation 0.1375
|
|
C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma
Week 6 (1 h post-dose 12)
|
0.865 Units per milliliter (U/mL)
Standard Deviation 0.1550
|
0.570 Units per milliliter (U/mL)
Standard Deviation 0.1190
|
|
C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma
Week 12 (Pre-dose 24)
|
0.362 Units per milliliter (U/mL)
Standard Deviation 0.1897
|
0.255 Units per milliliter (U/mL)
Standard Deviation 0.1108
|
|
C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma
Week 12 (1 h post-dose 24)
|
0.803 Units per milliliter (U/mL)
Standard Deviation 0.1906
|
0.531 Units per milliliter (U/mL)
Standard Deviation 0.1330
|
|
C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma
Week 12 (2 h post-dose 24)
|
0.613 Units per milliliter (U/mL)
Standard Deviation 0.2601
|
0.497 Units per milliliter (U/mL)
Standard Deviation 0.0635
|
|
C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma
Week 12 (4 h post-dose 24)
|
0.590 Units per milliliter (U/mL)
Standard Deviation 0.1803
|
0.497 Units per milliliter (U/mL)
Standard Deviation 0.0058
|
|
C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma
Week 12 (8 h post-dose 24)
|
0.643 Units per milliliter (U/mL)
Standard Deviation 0.0723
|
0.430 Units per milliliter (U/mL)
Standard Deviation 0.0458
|
SECONDARY outcome
Timeframe: Pre-dose and 1 h post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention periodPopulation: PK set consisted of all pariticipants in the safety set with no major deviations related to investigational product intake and evaluable PK profiles.
Concentration of Complement C4 in plasma was determined using an automated nephelometric assay.
Outcome measures
| Measure |
Treatment B (1000 U CINRYZE)
n=12 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment A (500 U CINRYZE)
n=12 Participants
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
Plasma Concentration of Complement C4
Week 1 (Pre-dose 1)
|
71.2 Milligram per liter (mg/L)
Standard Deviation 29.63
|
105.1 Milligram per liter (mg/L)
Standard Deviation 39.38
|
|
Plasma Concentration of Complement C4
Week 1 (1 h post-dose 1)
|
71.4 Milligram per liter (mg/L)
Standard Deviation 33.20
|
99.7 Milligram per liter (mg/L)
Standard Deviation 36.73
|
|
Plasma Concentration of Complement C4
Week 6 (Pre-dose 12)
|
121.3 Milligram per liter (mg/L)
Standard Deviation 41.50
|
97.3 Milligram per liter (mg/L)
Standard Deviation 37.26
|
|
Plasma Concentration of Complement C4
Week 6 (1 h post-dose 12)
|
111.7 Milligram per liter (mg/L)
Standard Deviation 41.75
|
88.0 Milligram per liter (mg/L)
Standard Deviation 27.75
|
|
Plasma Concentration of Complement C4
Week 12 (Pre-dose 24)
|
111.6 Milligram per liter (mg/L)
Standard Deviation 50.28
|
83.3 Milligram per liter (mg/L)
Standard Deviation 21.63
|
|
Plasma Concentration of Complement C4
Week 12 (1 h post-dose 24)
|
90.7 Milligram per liter (mg/L)
Standard Deviation 27.72
|
79.2 Milligram per liter (mg/L)
Standard Deviation 20.21
|
|
Plasma Concentration of Complement C4
Week 12 (2 h post-dose 24)
|
94.3 Milligram per liter (mg/L)
Standard Deviation 32.04
|
86.7 Milligram per liter (mg/L)
Standard Deviation 4.93
|
|
Plasma Concentration of Complement C4
Week 12 (4 h post-dose 24)
|
103.7 Milligram per liter (mg/L)
Standard Deviation 32.35
|
89.3 Milligram per liter (mg/L)
Standard Deviation 12.66
|
|
Plasma Concentration of Complement C4
Week 12 (8 h post-dose 24)
|
114.7 Milligram per liter (mg/L)
Standard Deviation 30.75
|
99.3 Milligram per liter (mg/L)
Standard Deviation 11.02
|
SECONDARY outcome
Timeframe: Pre-dose, 1 week post treatment (Week 13, Week 25) and 1 month post treatment follow-up (Week 28)Population: Safety set included all participants who received at least 1 dose of investigational product.
The presence of C1 INH antibodies in plasma samples was determined using a proprietary enzyme-linked-immunosorbent-assay. Number of participants with C1 INH Antibodies was reported.
Outcome measures
| Measure |
Treatment B (1000 U CINRYZE)
n=12 Participants
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment A (500 U CINRYZE)
n=12 Participants
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
Number of Participants With C1 Esterase Inhibitor (C1 INH) Antibodies in Plasma
|
0 Participants
|
0 Participants
|
Adverse Events
Treatment A (500 U CINRYZE)
Treatment B (1000 U CINRYZE)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment A (500 U CINRYZE)
n=12 participants at risk
Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks.
|
Treatment B (1000 U CINRYZE)
n=12 participants at risk
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks.
|
|---|---|---|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Musculoskeletal and connective tissue disorders
Coccydynia
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
8.3%
1/12 • Number of events 2 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Vascular disorders
Vascular pain
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Eye disorders
Eye pain
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Gastrointestinal disorders
Abdominal discomfort
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Gastrointestinal disorders
Abdominal pain
|
8.3%
1/12 • Number of events 2 • From start of study drug administration up to Week 25
|
16.7%
2/12 • Number of events 2 • From start of study drug administration up to Week 25
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Gastrointestinal disorders
Dental caries
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Gastrointestinal disorders
Diarrhoea
|
8.3%
1/12 • Number of events 2 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Gastrointestinal disorders
Nausea
|
8.3%
1/12 • Number of events 2 • From start of study drug administration up to Week 25
|
16.7%
2/12 • Number of events 2 • From start of study drug administration up to Week 25
|
|
Gastrointestinal disorders
Toothache
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 4 • From start of study drug administration up to Week 25
|
|
Gastrointestinal disorders
Vomiting
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
General disorders
Facial pain
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
General disorders
Fatigue
|
8.3%
1/12 • Number of events 9 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 9 • From start of study drug administration up to Week 25
|
|
General disorders
Infusion site pain
|
8.3%
1/12 • Number of events 19 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
General disorders
Pyrexia
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Congenital, familial and genetic disorders
Hereditary angioedema
|
75.0%
9/12 • Number of events 41 • From start of study drug administration up to Week 25
|
66.7%
8/12 • Number of events 25 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Gingivitis
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Infections and infestations
H1n1 influenza
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Hordeolum
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Nasopharyngitis
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
16.7%
2/12 • Number of events 3 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Paronychia
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Sinusitis
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Tinea pedis
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Tonsillitis
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Upper respiratory tract infection
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
16.7%
2/12 • Number of events 4 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Viral pharyngitis
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Infections and infestations
Viral upper respiratory tract infection
|
16.7%
2/12 • Number of events 3 • From start of study drug administration up to Week 25
|
50.0%
6/12 • Number of events 8 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Excoriation
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Face injury
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Fall
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Head injury
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Joint injury
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Lip injury
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Multiple injuries
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Post-Traumatic neck syndrome
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Injury, poisoning and procedural complications
Sunburn
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Metabolism and nutrition disorders
Decreased appetite
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Nervous system disorders
Dizziness
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Nervous system disorders
Headache
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
16.7%
2/12 • Number of events 5 • From start of study drug administration up to Week 25
|
|
Psychiatric disorders
Irritability
|
8.3%
1/12 • Number of events 7 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 7 • From start of study drug administration up to Week 25
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.7%
2/12 • Number of events 2 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
16.7%
2/12 • Number of events 2 • From start of study drug administration up to Week 25
|
|
Skin and subcutaneous tissue disorders
Angioedema
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
|
Skin and subcutaneous tissue disorders
Erythema marginatum
|
16.7%
2/12 • Number of events 12 • From start of study drug administration up to Week 25
|
16.7%
2/12 • Number of events 12 • From start of study drug administration up to Week 25
|
|
Skin and subcutaneous tissue disorders
Prurigo
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/12 • From start of study drug administration up to Week 25
|
8.3%
1/12 • Number of events 1 • From start of study drug administration up to Week 25
|
Additional Information
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