Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

Study Overview
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Brief Summary
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This is a prospective, international, multi-center, non-randomized, single arm, open-label, postmarketing study to investigate the formation of inhibitory anti-C1-INH antibodies in HAE subjects treated intravenously with Berinert. Individual treatment duration per subject is 9 months, irrespective of the number of treated attacks. All subjects will receive 20 IU Berinert/kg body weight per attack.

Official Title
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Prospective Open-label Uncontrolled Multi-center Post-marketing Study to Assess Inhibitory Antibody Formation in Subjects With Congenital C1-INH Deficiency and Acute Hereditary Angioedema (HAE) Attacks Treated With Berinert® , a C1-esterase Inhibitor

Conditions
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Hereditary Angioedema Types I and II

Intervention / Treatment
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* Biological: Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of congenital C1-INH deficiency (HAE type I or II) and assessed by the investigator to likely require intravenous (IV) Berinert treatment during the study period. Male or female, ≥ 12 years of age at the time of signing informed consent. Written informed consent for study participation obtained before undergoing any study specific procedures. Exclusion Criteria: Incurable malignancies in the last 6 months prior to study entry. Acquired angioedema due to C1-INH deficiency. All other types of angioedema not associated with C1-INH deficiency. Use of any C1-INH products other than Berinert within 30 days before the study, or planned use during the study. Immunization within 30 days prior to study entry. Autoimmune conditions requiring use of immunosuppressants during the study. Known or suspected hypersensitivity to C1-INH. Participation in any of the previous Berinert studies from which anti-C1-INH antibody results were submitted to the Food and Drug Administration.

Ages Eligible for Study
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Minimum Age: 12 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Berinert<br> | Biological: Berinert, lyophilizate for IV application containing 500 IU C1-INH to be reconstituted with 10 mL water for injection<br>* Individual treatment duration per subject is 9 months, irrespective of the number of treated attacks. Each attack that occurs in this time frame will be treated with 20 IU Berinert/kg body weight.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies | Subjects with no positive baseline result and at least one positive post-baseline result for inhibitory anti-C1-INH antibodies. | Baseline to approximately 9 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies | Subjects with at least one positive result for inhibitory or non-inhibitory anti-C1-INH antibodies. | Baseline to approximately 9 months |