A Phase 2 interventional study of CINRYZE in Hereditary Angioedema (HAE), sponsored by Shire. Completed at 11 sites in 3 countries. Open to participants aged 2 Years to 11 Years. Per ClinicalTrials.gov, last updated 2021-06-03.
Sponsored by Shire · Phase 2, Interventional, and Treatment
The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in children above and below 25 kg and less than 12 years of age with hereditary angioedema (HAE); and (3) to determine the safety and tolerability following IV administration of CINRYZE in this study population.
Each subject received CINRYZE for treatment of a single acute angioedema attack.
164 studies on the registry are indexed under Angioedema; 19 are open to participants now.
This study's enrollment of 9 is below the median of 44 across 111 interventional studies indexed under Angioedema.
Browse Angioedema studies →Shire is the lead sponsor of 346 studies on the registry; 2 are open to participants now.
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To be eligible for this protocol, subjects must:
Exclusion Criteria:
To be eligible for this protocol, subjects must not:
Single IV dose of 500 U CINRYZE
Biological: CINRYZE
Single IV dose of 1000 U CINRYZE
Biological: CINRYZE
Single IV dose of 1000 U CINRYZE
Biological: CINRYZE
Single IV dose of 1500 U CINRYZE
Biological: CINRYZE
Also known as: C1 inhibitor [human]
Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
Time frame: Within 4 hours following treatment
Time to Unequivocal Beginning of Relief of the Defining Attack Symptom
Time frame: Within 4 hours following treatment
Time to Complete Resolution of the Attack
Time frame: Within 1 week following treatment
Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations
Data was not reported due to change in planned analysis.
Time frame: Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8
Participants were not enrolled in the lower body weight category (10-25 kilograms \[kg\]) 1000 Units (U) dose group despite substantial recruitment efforts.
| Milestone | 500 U CINRYZE (10-25 kg Body Weight) | 1000 U CINRYZE (10-25 kg Body Weight) | 1000 U CINRYZE (>25 kg Body Weight) | 1500 U CINRYZE (>25 kg Body Weight) |
|---|---|---|---|---|
| Started | 3 | 0 | 3 | 3 |
| Completed | 3 | 0 | 3 | 3 |
| Not completed | 0 | 0 | 0 | 0 |
| participants | 500 U CINRYZE (10-25 kg Body Weight) | 1000 U CINRYZE (>25 kg Body Weight) | 1500 U CINRYZE (>25 kg Body Weight) |
|---|---|---|---|
| Yes | 3 | 3 | 3 |
| No | 0 | 0 | 0 |
| hours | 500 U CINRYZE (10-25 kg Body Weight) | 1000 U CINRYZE (>25 kg Body Weight) | 1500 U CINRYZE (>25 kg Body Weight) |
|---|---|---|---|
| Time to Unequivocal Beginning of Relief of the Defining Attack Symptom | 1.25 (0.25 to 1.75) | 0.25 (0.25 to 0.50) | 0.50 (0.25 to 2.50) |
| hours | 500 U CINRYZE (10-25 kg Body Weight) | 1000 U CINRYZE (>25 kg Body Weight) | 1500 U CINRYZE (>25 kg Body Weight) |
|---|---|---|---|
| Time to Complete Resolution of the Attack | 13.58 (11.48 to 37.35) | 10.00 (1.57 to 22.33) | 29.07 (1.58 to 102.33) |
Data was not reported due to change in planned analysis.
No measurements were reported for this outcome.
Collected over Adverse events were collected through 1 week following the dose of study drug.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 500 U CINRYZE (10-25 kg Body Weight) | — | 0/3 (0%) | 1/3 (33.3%) |
| 1000 U CINRYZE (>25 kg Body Weight) | — | 0/3 (0%) | 0/3 (0%) |
| 1500 U CINRYZE (>25 kg Body Weight) | — | 0/3 (0%) | 0/3 (0%) |
| Event | 500 U CINRYZE (10-25 kg Body Weight) | 1000 U CINRYZE (>25 kg Body Weight) | 1500 U CINRYZE (>25 kg Body Weight) |
|---|---|---|---|
| DiarrheaGastrointestinal disorders | 1/3 | 0/3 | 0/3 |
| NauseaGastrointestinal disorders | 1/3 | 0/3 | 0/3 |
| Age, Continuous(years) | 500 U CINRYZE (10-25 kg Body Weight) | 1000 U CINRYZE (>25 kg Body Weight) | 1500 U CINRYZE (>25 kg Body Weight) | Total |
|---|---|---|---|---|
| Median | 7 (6 to 9) | 9 (7 to 9) | 10 (8 to 11) | 9 (6 to 11) |
| Sex: Female, Male(Participants) | 500 U CINRYZE (10-25 kg Body Weight) | 1000 U CINRYZE (>25 kg Body Weight) | 1500 U CINRYZE (>25 kg Body Weight) | Total |
|---|---|---|---|---|
| Female | 3 | 3 | 2 | 8 |
| Male | 0 | 0 | 1 | 1 |
| Region of Enrollment(Participants) | 500 U CINRYZE (10-25 kg Body Weight) | 1000 U CINRYZE (>25 kg Body Weight) | 1500 U CINRYZE (>25 kg Body Weight) | Total |
|---|---|---|---|---|
| United States | 3 | 3 | 3 | 9 |
This study is completed, as verified in May 2021. You cannot join it, but the record below documents what was studied.
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