Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)

Study Overview
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Brief Summary
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The Icatibant Outcome Survey (IOS) is a prospective, observational disease registry designed to document the routine clinical outcomes over time in participants with angioedema treated with Firazyr® (icatibant) and/or Cinryze® (C1 inhibitor [human]) in countries where it is currently approved. The data collected will be used to evaluate the safety of Firazyr (icatibant) and Cinryze (C1 inhibitor [human]) in routine clinical practice and as a data source for post-marketing investigations.

Detailed Description
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The Icatibant Outcome Survey (IOS) is a multicenter, prospective, observational study for participants treated with Firazyr (icatibant) and/or Cinryze (C1 inhibitor [human]) in countries where it is currently approved. The entry of participants in the Icatibant Outcome Survey (IOS) is at the discretion of the physician and the participant and is not a pre-requisite for prescribing Firazyr (icatibant) or Cinryze (C1 inhibitor [human]).

Official Title
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Icatibant Outcome Survey (IOS) Registry

Conditions
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Hereditary Angioedema (HAE)

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of at least 1 of the following: Hereditary angioedema (HAE) type I or II HAE with normal C1 inhibitor ACE-I-induced angioedema Non-histaminergic idiopathic angioedema Acquired angioedema. Signed and dated written informed consent from the participant or, for participants aged less than(<)18 years (or as per local regulation, such as <16 years in the United Kingdom [UK]), parent and/or participants legally authorized representative (LAR), and assent of the minor where applicable. At sites only participating in the drug registry, participants must have taken at least 1 dose of Firazyr (Icatibant) or Cinryze (C1 inhibitor [human]). Enrolled participants in Germany taking Firazyr (Icatibant) or Cinryze (C1 inhibitor [human]) will only use the respective product in accordance with the product label. Exclusion Criteria: Participants enrolled in clinical trials where the product is blinded or where the product under investigation is for the treatment of HAE, ACE-I-induced angioedema, non-histaminergic idiopathic angioedema, or acquired angioedema. Participants enrolled in another Shire-sponsored registry involving products for the treatment of HAE, ACE-I-induced angioedema, non-histaminergic idiopathic angioedema, or acquired angioedema. An exception applies to participants enrolled in the Shire lanadelumab ENABLE study.
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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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Participants with hereditary angioedema (HAE)<br>All participants with hereditary angioedema (HAE) who are administered Cinryze (C1 inhibitor [human]) or Firazyr (Icatibant) for the treatment or prevention of angioedema attacks in routine clinical practice will be included into the study. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Cardiac Ischemia Events in Participants Predisposed to Cardiac Ischemia Events With Concomitant Firazyr (Icatibant) Administration | Incidence of cardiac ischemia events in participants predisposed to cardiac ischemia events with concomitant Firazyr (Icatibant) administration will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Hypotension for Firazyr (Icatibant) | Incidence of hypotension for Firazyr (Icatibant) will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Swelling of Mucous Membranes for Firazyr (Icatibant) | Incidence of swelling of mucous membranes for Firazyr (Icatibant) will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Bronchoconstriction for Firazyr (Icatibant) | Incidence of bronchoconstriction for Firazyr (Icatibant) will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Aggravation of Pain for Firazyr (Icatibant) | Incidence of aggravation of pain for Firazyr (Icatibant) will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Sexual Hormones Level Measurements- Tanner Staging for Firazyr (Icatibant) | Effects on sexual maturation in pubertal adolescents will be measured using Tanner staging (pubic hair stage and genital breast stage) for Firazyr (Icatibant). | From enrollment through study participation (Approximately 13 years) | 
| Time to Complete Resolution of the Firazyr (Icatibant)-Treated Laryngeal Attacks | Time to complete resolution of the laryngeal attacks will be assessed. It is defined as the time between the first injection of treatment and the complete resolution of all symptoms. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Adverse Events (AE) Related to Firazyr (Icatibant)-Treated Laryngeal Attacks | An AE is defined as any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Adverse Drug Reactions (ADR) for Firazyr (Icatibant) | An ADR is a response to a medicinal product that is noxious and unintended and that occurs at doses normally used in man for prophylaxis, diagnosis, and treatment of disease or for the restoration, correction, or modification of physiological function. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Serious Adverse Events (SAE) for Firazyr (Icatibant) | An AE or ADR that meets 1 or more of the following criteria or outcomes is classified as an SAE whether considered to be related to the pharmaceutical product or not: death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; a persistent or significant disability or incapacity; a congenital anomaly or birth defect; important medical events. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Pregnancy and Lactation Events During Firazyr (Icatibant) Exposure | The incidence of pregnancy or lactation events coinciding with exposure to Firazyr (Icatibant) will be summarized by angioedema treatment and subgroup. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Adverse Events (AE) for Cinryze (C1 Inhibitor [Human]) | An AE is defined as any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in the registry, whether or not considered product-related. This includes an exacerbation of a pre-existing condition. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Adverse Drug Reactions (ADR) for Cinryze (C1 Inhibitor [Human]) | An ADR is a response to a medicinal product that is noxious and unintended and that occurs at doses normally used in man for prophylaxis, diagnosis, and treatment of disease or for the restoration, correction, or modification of physiological function. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Serious Adverse Events (SAE) for Cinryze (C1 Inhibitor [Human]) | An AE or ADR that meets 1 or more of the following criteria or outcomes is classified as an SAE whether considered to be related to the pharmaceutical product or not: death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; a persistent or significant disability or incapacity; a congenital anomaly or birth defect; important medical events. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Thrombotic or Thromboembolic Events for Cinryze (C1 Inhibitor [Human]) | Thrombotic or thromboembolic events will be reported as SAEs and will include, but are not limited to, established diagnoses of any of the following: renal allograft arterial or venous thrombosis; deep vein thrombosis; myocardial infarction; pulmonary embolism; Ischemic cerebrovascular accident (stroke)- cerebrovascular accident exclusive of cerebrovascular hemorrhage (subarachnoid or subdural hemorrhage); any large vessel thrombosis; thrombophlebitis; catheter-related thrombotic events (including clotted dialysis access grafts) will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Incidence of Pregnancy and Lactation Events During Cinryze (C1 Inhibitor [Human]) Exposure | The incidence of pregnancy or lactation events coinciding with exposure to Cinryze (C1 inhibitor [human]) will be summarized by angioedema treatment and subgroup. | From enrollment through study participation (Approximately 13 years) | 
| Drug Exposure Data for Cinryze (C1 Inhibitor [Human]) | Drug exposure data for Cinryze (C1 inhibitor [human]) for prophylaxis, pre-procedural, and acute treatments will be reported. | From enrollment through study participation (Approximately 13 years) | 
| Frequency of Hereditary Angioedema (HAE) Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human]) | Frequency of HAE attacks in participants treated with Cinryze (C1 inhibitor [human]) will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Severity of Hereditary Angioedema Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human]) | Severity of HAE attacks in participants treated with Cinryze (C1 inhibitor [human]) will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Anatomic Location of Hereditary Angioedema Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human]) | Anatomic location of HAE attacks in participants treated with Cinryze (C1 inhibitor [human]) will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Outcome of Severe or Laryngeal Hereditary Angioedema Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human]) | Outcome of severe or laryngeal HAE attacks in participants treated with Cinryze (C1 inhibitor [human]) will be assessed. | From enrollment through study participation (Approximately 13 years) | 
| Outcome of Hereditary Angioedema Attacks for Treatment With Cinryze (C1 Inhibitor [Human]) | Outcome of HAE attacks for treatment with Cinryze (C1 inhibitor [human]) which was initiated more than 4 hours after onset of the attack will be reported. | From enrollment through study participation (Approximately 13 years) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to Treatment For Attack | Time to treatment for attack will be assessed. It is defined as the time between the onset of the attack and the first injection of treatment. | From enrollment through study participation (Approximately 13 years) | 
| Time to Complete Resolution of Attack | Time to complete resolution of attack will be assessed. It is defined as the time between the first injection of treatment and the complete resolution of all symptoms. | From enrollment through study participation (Approximately 13 years) | 
| Total Duration of Attack | Total duration of attack will be assessed. It is defined as the time between the onset of the attack and the complete resolution of all symptoms | From enrollment through study participation (Approximately 13 years) | 
| Hereditary Angioedema-Treated Attacks | The frequency, severity, and affected sites of HAE-treated attacks will be reported. | From enrollment through study participation (Approximately 13 years) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hereditary angioedema