Phase 2
N=9
CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12
Hereditary Angioedema (HAE)
Bottom Line
View on ClinicalTrials.gov: NCT01095510 ↗Enrolled (actual)
9
Serious AEs
0.0%
Results posted
Jul 2014
Primary outcome: Primary: Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom — 3; 3; 3; 0 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- CINRYZE (Biological)
- Age
- Pediatric · 2+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Apr 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom |
3; 3; 3; 0; 0; 0 | — |
| SECONDARY Time to Unequivocal Beginning of Relief of the Defining Attack Symptom |
1.25; 0.25; 0.50 | — |
| SECONDARY Time to Complete Resolution of the Attack |
13.58; 10.00; 29.07 | — |
| SECONDARY Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations |
— | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
To be eligible for this protocol, subjects must:
- Be at least 10 kg of body weight.
- Have a confirmed diagnosis of HAE.
- Have an acute HAE attack and be able to initiate treatment within 8 hours after onset of symptoms.
Exclusion Criteria
To be eligible for this protocol, subjects must not:
- Have any active infectious illness.
- Have had a prior HAE attack and/or received any C1 INH product within 7 days prior to dosing with study drug.
- Have received therapy with antifibrinolytics (e.g., tranexamic acid), androgens (e.g., danazol, oxandrolone, stanozolol, or testosterone), ecallantide (Kalbitor®), or icatibant (Firazyr®) within 7 days prior to dosing with study drug.
- Have a history of allergic reaction to C1 INH products, including CINRYZE (or any of the components of CINRYZE), or other blood products.
- Have participated in any other investigational drug evaluation within 30 days prior to dosing with study drug, or have previously received treatment with CINRYZE in this study at any time.
Data sourced from ClinicalTrials.gov (NCT01095510). 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.