Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema

Study Overview
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Brief Summary
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The primary objective of this study is: assess the safety and tolerability of ecallantide in paediatric patients for acute attacks of HAE The secondary objectives are: evaluate the pharmacokinetic profile of ecallantide in paediatric patients treated for acute attacks of HAE assess the efficacy of ecallantide in paediatric patients treated for moderate to severe acute attacks of HAE

Official Title
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A 3-Part Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Subcutaneous Ecallantide in Prepubertal Paediatric Patients Experiencing Acute Attacks of Hereditary Angioedema (HAE)

Conditions
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Hereditary Angioedema (HAE)

Intervention / Treatment
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* Biological: ecallantide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female from 2 years of age and prepubertal Physician diagnosis and history of HAE (Type I or II) including laboratory results showing C1-1NH activity below the lower limit of normal or up to 15% above the lower limit of the normal range Present at the site with moderate to severe signs and symptoms of an acute attack of HAE within 8 hours of recognition of the onset of the attack. Spontaneous resolution of the attack must not have begun before the administration of study drug. Must have signed informed consent by parent or caregiver. Exclusion Criteria: <2 years of age or have reached puberty Received treatment with ecallantide within previous 72 hours Received an investigational drug or device, other than ecallantide, within 30 days prior to the screening visit Pharyngeal/laryngeal symptoms Mild attacks including mild edema of the extremities and mild abdominal attacks Are unable or unwilling to give informed consent (parent or caregiver) Any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 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: Randomized
Intervention Model: Single Group Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: ecallantide<br> | Biological: ecallantide<br>* 10 - 30mg subcutaneous injection.<br>|
| Placebo Comparator: placebo<br>phosphate buffered saline | Biological: ecallantide<br>* 10 - 30mg subcutaneous injection.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| safety and tolerability of ecallantide |  | 4 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| evaluate the PK profile of ecallantide |  | 4 years | 
| assess the efficacy of ecallantide |  | 4 years |