CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

Study Overview
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Brief Summary
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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.

Detailed Description
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Each subject received CINRYZE for treatment of a single acute angioedema attack.

Official Title
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Open-Label, Single-Dose Study to Evaluate the Response and Pharmacokinetics/Pharmacodynamics of Different Doses of CINRYZE® [C1 Inhibitor (Human)] For Treatment of Acute Angioedema Attacks in Children Less Than 12 Years of Age With Hereditary Angioedema

Conditions
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Hereditary Angioedema (HAE)

Intervention / Treatment
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* Biological: CINRYZE


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 11 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 500 U CINRYZE (10-25 kg body weight)<br>Single IV dose of 500 U CINRYZE | Biological: CINRYZE<br> <br> * Other names: C1 inhibitor [human];|
| Experimental: 1000 U CINRYZE (10-25 kg body weight)<br>Single IV dose of 1000 U CINRYZE | Biological: CINRYZE<br> <br> * Other names: C1 inhibitor [human];|
| Experimental: 1000 U CINRYZE (>25 kg body weight)<br>Single IV dose of 1000 U CINRYZE | Biological: CINRYZE<br> <br> * Other names: C1 inhibitor [human];|
| Experimental: 1500 U CINRYZE (>25 kg body weight)<br>Single IV dose of 1500 U CINRYZE | Biological: CINRYZE<br> <br> * Other names: C1 inhibitor [human];|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom |  | Within 4 hours following treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to Unequivocal Beginning of Relief of the Defining Attack Symptom |  | Within 4 hours following treatment | 
| Time to Complete Resolution of the Attack |  | 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. | Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CINRYZE, C1 INH, HAE, C1 inhibitor, Pediatric, PK/PD