Trial Outcomes & Findings for CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12 (NCT NCT01095510)
NCT ID: NCT01095510
Last Updated: 2021-06-03
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
9 participants
Primary outcome timeframe
Within 4 hours following treatment
Results posted on
2021-06-03
Participant Flow
Participants were not enrolled in the lower body weight category (10-25 kilograms \[kg\]) 1000 Units (U) dose group despite substantial recruitment efforts.
Of 12 participants screened, 9 participants were enrolled and treated. The reason for 3 participants were screen failures as they did not meet the inclusion criteria.
Participant milestones
| Measure |
500 U CINRYZE (10-25 kg Body Weight)
Single intravenous (IV) dose of 500 U CINRYZE
|
1000 U CINRYZE (10-25 kg Body Weight)
Single IV dose of 1000 U CINRYZE
|
1000 U CINRYZE (>25 kg Body Weight)
Single IV dose of 1000 U CINRYZE
|
1500 U CINRYZE (>25 kg Body Weight)
Single IV dose of 1500 U CINRYZE
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
3
|
0
|
3
|
3
|
|
Overall Study
COMPLETED
|
3
|
0
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12
Baseline characteristics by cohort
| Measure |
500 U CINRYZE (10-25 kg Body Weight)
n=3 Participants
Single IV dose of 500 U CINRYZE
|
1000 U CINRYZE (>25 kg Body Weight)
n=3 Participants
Single IV dose of 1000 U CINRYZE
|
1500 U CINRYZE (>25 kg Body Weight)
n=3 Participants
Single IV dose of 1500 U CINRYZE
|
Total
n=9 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
7 years
n=5 Participants
|
9 years
n=7 Participants
|
10 years
n=5 Participants
|
9 years
n=4 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Within 4 hours following treatmentPopulation: Intent-to-treat efficacy (ITT-E) population included all participants with baseline and at least one post-infusion investigator assessment of the hereditary angioedema (HAE) attack.
Outcome measures
| Measure |
500 U CINRYZE (10-25 kg Body Weight)
n=3 Participants
Single IV dose of 500 U CINRYZE
|
1000 U CINRYZE (>25 kg Body Weight)
n=3 Participants
Single IV dose of 1000 U CINRYZE
|
1500 U CINRYZE (>25 kg Body Weight)
n=3 Participants
Single IV dose of 1500 U CINRYZE
|
|---|---|---|---|
|
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
Yes
|
3 participants
|
3 participants
|
3 participants
|
|
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
No
|
0 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Within 4 hours following treatmentPopulation: ITT-E population
Outcome measures
| Measure |
500 U CINRYZE (10-25 kg Body Weight)
n=3 Participants
Single IV dose of 500 U CINRYZE
|
1000 U CINRYZE (>25 kg Body Weight)
n=3 Participants
Single IV dose of 1000 U CINRYZE
|
1500 U CINRYZE (>25 kg Body Weight)
n=3 Participants
Single IV dose of 1500 U CINRYZE
|
|---|---|---|---|
|
Time to Unequivocal Beginning of Relief of the Defining Attack Symptom
|
1.25 hours
Interval 0.25 to 1.75
|
0.25 hours
Interval 0.25 to 0.5
|
0.50 hours
Interval 0.25 to 2.5
|
SECONDARY outcome
Timeframe: Within 1 week following treatmentPopulation: ITT-E population.
Outcome measures
| Measure |
500 U CINRYZE (10-25 kg Body Weight)
n=3 Participants
Single IV dose of 500 U CINRYZE
|
1000 U CINRYZE (>25 kg Body Weight)
n=3 Participants
Single IV dose of 1000 U CINRYZE
|
1500 U CINRYZE (>25 kg Body Weight)
n=3 Participants
Single IV dose of 1500 U CINRYZE
|
|---|---|---|---|
|
Time to Complete Resolution of the Attack
|
13.58 hours
Interval 11.48 to 37.35
|
10.00 hours
Interval 1.57 to 22.33
|
29.07 hours
Interval 1.58 to 102.33
|
SECONDARY outcome
Timeframe: Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8Population: No participant agreed to obtain pharmacokinetic (PK) blood sampling for antigenic and functional C1 INH levels. Hence, it was planned not to be analyzed.
Data was not reported due to change in planned analysis.
Outcome measures
Outcome data not reported
Adverse Events
500 U CINRYZE (10-25 kg Body Weight)
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
1000 U CINRYZE (>25 kg Body Weight)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
1500 U CINRYZE (>25 kg Body Weight)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
500 U CINRYZE (10-25 kg Body Weight)
n=3 participants at risk
Single IV dose of 500 U CINRYZE
|
1000 U CINRYZE (>25 kg Body Weight)
n=3 participants at risk
Single IV dose of 1000 U CINRYZE
|
1500 U CINRYZE (>25 kg Body Weight)
n=3 participants at risk
Single IV dose of 1500 U CINRYZE
|
|---|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
1/3 • Adverse events were collected through 1 week following the dose of study drug.
|
0.00%
0/3 • Adverse events were collected through 1 week following the dose of study drug.
|
0.00%
0/3 • Adverse events were collected through 1 week following the dose of study drug.
|
|
Gastrointestinal disorders
Nausea
|
33.3%
1/3 • Adverse events were collected through 1 week following the dose of study drug.
|
0.00%
0/3 • Adverse events were collected through 1 week following the dose of study drug.
|
0.00%
0/3 • Adverse events were collected through 1 week following the dose of study drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Clinical Trial Agreement. Most restrictive provision - PI will not publish results until after first of: multicenter publication is published or 24 months from study end. Thereafter, PI may publish his results. PI must provide copy of proposed publication to Sponsor for pre-review. If Sponsor requests, PI must delete Sponsor confidential information before publication and/or delay publication for 60 days so Sponsor can file for patents or take other action to protect its patent rights.
- Publication restrictions are in place
Restriction type: OTHER