C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)

Study Overview
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Brief Summary
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Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of involvement of the larynx.The planned extension study is designed to enrol subjects that participated in the pivotal study in order to provide them with C1-INH for treatment of acute HAE attacks for 24 months or until the licensing procedure for C1-INH is finalized, whatever comes first.

Official Title
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Open-label Extension Study of CE1145 (Human Pasteurized C1 Esterase Inhibitor Concentrate) in Subjects With Congenital C1-INH Deficiency and Acute HAE Attacks

Conditions
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Hereditary Angioedema

Intervention / Treatment
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* Drug: C1 Esterase Inhibitor


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Documented congenital C1-INH deficiency Acute HAE attack Participation in base study CE1145_3001 (NCT00168103) Key Exclusion Criteria: Acquired angioedema Treatment with any other investigational drug besides CE1145 in the last 30 days before study entry

Ages Eligible for Study
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Minimum Age: 6 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: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: C1 Esterase Inhibitor<br> | Drug: C1 Esterase Inhibitor<br>* Lyophilisate containing approximately 500 U C1-INH to be reconstituted with 10 mL water for injection; Single Dose: 20 U/kg b.w. iv<br>* Other names: Berinert P;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population) | The start of symptom relief was determined by subject self-assessment. | Up to 24 h after start of study treatment | 
| Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population) | The start of symptom relief was determined by subject self-assessment. | Up to 24 h after start of study treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to Complete Resolution of All HAE Symptoms (ITT Subject Population) | Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card. | Up to Day 9 following an attack | 
| Time to Complete Resolution of All HAE Symptoms (ITT Attack Population) | Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card. | Up to Day 9 following an attack | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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C1 Inhibitor, Hereditary angioedema, Acute HAE attack