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Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)

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ClinicalTrials.gov Identifier: NCT01034969
Recruitment Status : Recruiting
First Posted : December 18, 2009
Last Update Posted : June 4, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

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.

Condition or disease
Hereditary Angioedema (HAE)

Detailed Description:
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]).

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Study Type : Observational
Estimated Enrollment : 3000 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Icatibant Outcome Survey (IOS) Registry
Actual Study Start Date : July 10, 2009
Estimated Primary Completion Date : May 24, 2023
Estimated Study Completion Date : May 24, 2023


Group/Cohort
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.



Primary Outcome Measures :
  1. Incidence of Cardiac Ischemia Events in Participants Predisposed to Cardiac Ischemia Events With Concomitant Firazyr (Icatibant) Administration [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Incidence of cardiac ischemia events in participants predisposed to cardiac ischemia events with concomitant Firazyr (Icatibant) administration will be assessed.

  2. Incidence of Hypotension for Firazyr (Icatibant) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Incidence of hypotension for Firazyr (Icatibant) will be assessed.

  3. Incidence of Swelling of Mucous Membranes for Firazyr (Icatibant) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Incidence of swelling of mucous membranes for Firazyr (Icatibant) will be assessed.

  4. Incidence of Bronchoconstriction for Firazyr (Icatibant) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Incidence of bronchoconstriction for Firazyr (Icatibant) will be assessed.

  5. Incidence of Aggravation of Pain for Firazyr (Icatibant) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Incidence of aggravation of pain for Firazyr (Icatibant) will be assessed.

  6. Sexual Hormones Level Measurements- Tanner Staging for Firazyr (Icatibant) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Effects on sexual maturation in pubertal adolescents will be measured using Tanner staging (pubic hair stage and genital breast stage) for Firazyr (Icatibant).

  7. Time to Complete Resolution of the Firazyr (Icatibant)-Treated Laryngeal Attacks [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  8. Incidence of Adverse Events (AE) Related to Firazyr (Icatibant)-Treated Laryngeal Attacks [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  9. Incidence of Adverse Drug Reactions (ADR) for Firazyr (Icatibant) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  10. Incidence of Serious Adverse Events (SAE) for Firazyr (Icatibant) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  11. Incidence of Pregnancy and Lactation Events During Firazyr (Icatibant) Exposure [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    The incidence of pregnancy or lactation events coinciding with exposure to Firazyr (Icatibant) will be summarized by angioedema treatment and subgroup.

  12. Incidence of Adverse Events (AE) for Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  13. Incidence of Adverse Drug Reactions (ADR) for Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  14. Incidence of Serious Adverse Events (SAE) for Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  15. Incidence of Thrombotic or Thromboembolic Events for Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  16. Incidence of Pregnancy and Lactation Events During Cinryze (C1 Inhibitor [Human]) Exposure [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    The incidence of pregnancy or lactation events coinciding with exposure to Cinryze (C1 inhibitor [human]) will be summarized by angioedema treatment and subgroup.

  17. Drug Exposure Data for Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Drug exposure data for Cinryze (C1 inhibitor [human]) for prophylaxis, pre-procedural, and acute treatments will be reported.

  18. Frequency of Hereditary Angioedema (HAE) Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Frequency of HAE attacks in participants treated with Cinryze (C1 inhibitor [human]) will be assessed.

  19. Severity of Hereditary Angioedema Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Severity of HAE attacks in participants treated with Cinryze (C1 inhibitor [human]) will be assessed.

  20. Anatomic Location of Hereditary Angioedema Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Anatomic location of HAE attacks in participants treated with Cinryze (C1 inhibitor [human]) will be assessed.

  21. Outcome of Severe or Laryngeal Hereditary Angioedema Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    Outcome of severe or laryngeal HAE attacks in participants treated with Cinryze (C1 inhibitor [human]) will be assessed.

  22. Outcome of Hereditary Angioedema Attacks for Treatment With Cinryze (C1 Inhibitor [Human]) [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.


Secondary Outcome Measures :
  1. Time to Treatment For Attack [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  2. Time to Complete Resolution of Attack [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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.

  3. Total Duration of Attack [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    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

  4. Hereditary Angioedema-Treated Attacks [ Time Frame: From enrollment through study participation (Approximately 13 years) ]
    The frequency, severity, and affected sites of HAE-treated attacks will be reported.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants with Type I or II HAE, and, where applicable, with angiotensin-converting enzyme inhibitor (ACE-I)-induced angioedema, non-histaminergic idiopathic angioedema, or acquired angioedema, irrespective of treatment and/or other treatments and also participants who have taken at least 1 dose of Firazyr (Icatibant) or Cinryze (C1 inhibitor [human]) will be included in this study.
Criteria

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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01034969


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

  Show 70 Study Locations
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Shire

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01034969     History of Changes
Other Study ID Numbers: JE049-5134
First Posted: December 18, 2009    Key Record Dates
Last Update Posted: June 4, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
URL: https://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Shire:
Hereditary angioedema

Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Icatibant
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Inflammatory Agents
Antirheumatic Agents
Bradykinin B2 Receptor Antagonists
Bradykinin Receptor Antagonists
Molecular Mechanisms of Pharmacological Action
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors