Phase 3
N=12
Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema
Hereditary Angioedema (HAE)
Bottom Line
View on ClinicalTrials.gov: NCT02052141 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Aug 2018
Primary outcome: Primary: Normalized Number of Angioedema Attacks Per Month in a Treatment Period — 1.2; 0.7 Angioedema attacks per month — p=0.03
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- CINRYZE 500 (Biological); CINRYZE 1000 (Biological)
- Age
- Pediatric · 6+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- May 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Normalized Number of Angioedema Attacks Per Month in a Treatment Period |
1.2; 0.7 | 0.03 sig |
| SECONDARY Cumulative Attack-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period |
2.0; 1.4 | 0.05 |
| SECONDARY Cumulative Daily-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period |
4.1; 2.2 | 0.04 sig |
| SECONDARY Normalized Number of Angioedema Attacks Per Month Requiring Acute Treatment in a Treatment Period |
0.7; 0.4 | 0.07 |
| SECONDARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Dose Group |
10; 11 | — |
| SECONDARY Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen |
0.0945; 0.0736; 0.1819; 0.2084; 0.0965; 0.1068 | — |
| SECONDARY C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma |
0.290; 0.210; 0.575; 0.725; 0.297; 0.336 | — |
| SECONDARY Plasma Concentration of Complement C4 |
105.1; 71.2; 99.7; 71.4; 97.3; 121.3 | — |
| SECONDARY Number of Participants With C1 Esterase Inhibitor (C1 INH) Antibodies in Plasma |
0; 0 | — |
Summary
Primary Objective - To assess the relative efficacy of two dose levels of CINRYZE (500 Units and 1000 Units) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of age with hereditary angioedema (HAE).
Secondary Objectives - To assess the safety and tolerability, characterize the pharmacokinetics (PK) and pharmacodynamics (PD), and assess the immunogenicity of two dose levels of CINRYZE administered by IV injection in children 6 to 11 years of age with HAE.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of Type I or Type II HAE.
- History of angioedema attacks.
Exclusion Criteria
- History of bleeding or clotting abnormality.
- Diagnosis of acquired angioedema or known to have C1 INH antibodies.
- History of allergic reaction to C1 esterase inhibitor or other blood products.
- Receipt of any experimental agents other than those required for prevention or treatment of angioedema attacks within 30 days prior to screening.
Data sourced from ClinicalTrials.gov (NCT02052141). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.