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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01034969




Registration number
NCT01034969
Ethics application status
Date submitted
17/12/2009
Date registered
18/12/2009
Date last updated
4/06/2019

Titles & IDs
Public title
Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)
Scientific title
Icatibant Outcome Survey (IOS) Registry
Secondary ID [1] 0 0
JE049-5134
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema (HAE) 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Other blood disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Participants with hereditary angioedema (HAE) - 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.

Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [2] 0 0
Incidence of Hypotension for Firazyr (Icatibant) - Incidence of hypotension for Firazyr (Icatibant) will be assessed.
Timepoint [2] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [3] 0 0
Incidence of Swelling of Mucous Membranes for Firazyr (Icatibant) - Incidence of swelling of mucous membranes for Firazyr (Icatibant) will be assessed.
Timepoint [3] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [4] 0 0
Incidence of Bronchoconstriction for Firazyr (Icatibant) - Incidence of bronchoconstriction for Firazyr (Icatibant) will be assessed.
Timepoint [4] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [5] 0 0
Incidence of Aggravation of Pain for Firazyr (Icatibant) - Incidence of aggravation of pain for Firazyr (Icatibant) will be assessed.
Timepoint [5] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [6] 0 0
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).
Timepoint [6] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [7] 0 0
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.
Timepoint [7] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [8] 0 0
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.
Timepoint [8] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [9] 0 0
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.
Timepoint [9] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [10] 0 0
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.
Timepoint [10] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [11] 0 0
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.
Timepoint [11] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [12] 0 0
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.
Timepoint [12] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [13] 0 0
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.
Timepoint [13] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [14] 0 0
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.
Timepoint [14] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [15] 0 0
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.
Timepoint [15] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [16] 0 0
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.
Timepoint [16] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [17] 0 0
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.
Timepoint [17] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [18] 0 0
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.
Timepoint [18] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [19] 0 0
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.
Timepoint [19] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [20] 0 0
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.
Timepoint [20] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [21] 0 0
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.
Timepoint [21] 0 0
From enrollment through study participation (Approximately 13 years)
Primary outcome [22] 0 0
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.
Timepoint [22] 0 0
From enrollment through study participation (Approximately 13 years)
Secondary outcome [1] 0 0
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.
Timepoint [1] 0 0
From enrollment through study participation (Approximately 13 years)
Secondary outcome [2] 0 0
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.
Timepoint [2] 0 0
From enrollment through study participation (Approximately 13 years)
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
From enrollment through study participation (Approximately 13 years)
Secondary outcome [4] 0 0
Hereditary Angioedema-Treated Attacks - The frequency, severity, and affected sites of HAE-treated attacks will be reported.
Timepoint [4] 0 0
From enrollment through study participation (Approximately 13 years)

Eligibility
Key inclusion criteria
1. 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.

2. 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.

3. At sites only participating in the drug registry, participants must have taken at
least 1 dose of Firazyr (Icatibant) or Cinryze (C1 inhibitor [human]).

4. Enrolled participants in Germany taking Firazyr (Icatibant) or Cinryze (C1 inhibitor
[human]) will only use the respective product in accordance with the product label.
Minimum age
No limit
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. 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.

2. 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.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA
Recruitment hospital [1] 0 0
Campbelltown Hospital - Campbelltown
Recruitment hospital [2] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
2560 - Campbelltown
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment outside Australia
Country [1] 0 0
Austria
State/province [1] 0 0
Steiermark
Country [2] 0 0
Brazil
State/province [2] 0 0
Sao Paulo
Country [3] 0 0
Czechia
State/province [3] 0 0
Brno
Country [4] 0 0
Denmark
State/province [4] 0 0
Odense
Country [5] 0 0
France
State/province [5] 0 0
Bas-Rhin
Country [6] 0 0
France
State/province [6] 0 0
Calvados
Country [7] 0 0
France
State/province [7] 0 0
Haute-Garonne
Country [8] 0 0
France
State/province [8] 0 0
Nord
Country [9] 0 0
France
State/province [9] 0 0
Angers Cedex 10
Country [10] 0 0
France
State/province [10] 0 0
Bordeaux
Country [11] 0 0
France
State/province [11] 0 0
Grenoble
Country [12] 0 0
France
State/province [12] 0 0
Le Mans cedex 9
Country [13] 0 0
France
State/province [13] 0 0
Lyon
Country [14] 0 0
France
State/province [14] 0 0
Montpellier
Country [15] 0 0
France
State/province [15] 0 0
Nancy
Country [16] 0 0
France
State/province [16] 0 0
Nantes
Country [17] 0 0
France
State/province [17] 0 0
Nice
Country [18] 0 0
France
State/province [18] 0 0
Niort
Country [19] 0 0
France
State/province [19] 0 0
Paris
Country [20] 0 0
France
State/province [20] 0 0
Reims
Country [21] 0 0
France
State/province [21] 0 0
Saint-Etienne
Country [22] 0 0
Germany
State/province [22] 0 0
Bayern
Country [23] 0 0
Germany
State/province [23] 0 0
Hessen
Country [24] 0 0
Germany
State/province [24] 0 0
Nordrhein-Westfalen
Country [25] 0 0
Germany
State/province [25] 0 0
Rheinland-Pfalz
Country [26] 0 0
Germany
State/province [26] 0 0
Sachsen
Country [27] 0 0
Germany
State/province [27] 0 0
Berlin
Country [28] 0 0
Germany
State/province [28] 0 0
Mörfelden-Walldorf
Country [29] 0 0
Germany
State/province [29] 0 0
Ulm
Country [30] 0 0
Greece
State/province [30] 0 0
Attiki
Country [31] 0 0
Greece
State/province [31] 0 0
Thessaloniki
Country [32] 0 0
Israel
State/province [32] 0 0
Haifa
Country [33] 0 0
Israel
State/province [33] 0 0
Petach Tikva
Country [34] 0 0
Israel
State/province [34] 0 0
Ramat-Gan
Country [35] 0 0
Israel
State/province [35] 0 0
Tel Aviv
Country [36] 0 0
Italy
State/province [36] 0 0
Cagliari
Country [37] 0 0
Italy
State/province [37] 0 0
Campania
Country [38] 0 0
Italy
State/province [38] 0 0
Lombardia
Country [39] 0 0
Italy
State/province [39] 0 0
Puglia
Country [40] 0 0
Italy
State/province [40] 0 0
Sardegna
Country [41] 0 0
Italy
State/province [41] 0 0
Padova
Country [42] 0 0
Italy
State/province [42] 0 0
Palermo
Country [43] 0 0
Spain
State/province [43] 0 0
A Coruña
Country [44] 0 0
Spain
State/province [44] 0 0
Alicante
Country [45] 0 0
Spain
State/province [45] 0 0
Barcelona
Country [46] 0 0
Spain
State/province [46] 0 0
Gijon
Country [47] 0 0
Spain
State/province [47] 0 0
Jaen
Country [48] 0 0
Spain
State/province [48] 0 0
L'Hospitalet de Llobregat
Country [49] 0 0
Spain
State/province [49] 0 0
Lleida
Country [50] 0 0
Spain
State/province [50] 0 0
Logrono
Country [51] 0 0
Spain
State/province [51] 0 0
Madrid
Country [52] 0 0
Spain
State/province [52] 0 0
Sevilla
Country [53] 0 0
Spain
State/province [53] 0 0
Valencia
Country [54] 0 0
Spain
State/province [54] 0 0
Vigo
Country [55] 0 0
Sweden
State/province [55] 0 0
Jönköping
Country [56] 0 0
United Kingdom
State/province [56] 0 0
Oxfordshire
Country [57] 0 0
United Kingdom
State/province [57] 0 0
York
Country [58] 0 0
United Kingdom
State/province [58] 0 0
Cardiff
Country [59] 0 0
United Kingdom
State/province [59] 0 0
London
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Manchester
Country [61] 0 0
United Kingdom
State/province [61] 0 0
Newcastle Upon Tyne
Country [62] 0 0
United Kingdom
State/province [62] 0 0
Plymouth

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Shire
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/show/NCT01034969
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Shire
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Shire Contact
Address 0 0
Country 0 0
Phone 0 0
+1 866 842 5335
Fax 0 0
Email 0 0
ClinicalTransparency@shire.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT01034969