Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)

Study at a glance

From the registry record, last updated by the sponsor on 28 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title

Icatibant Outcome Survey (IOS) Registry

ClinicalTrials.gov ID

NCT01034969

Acronym / sponsor ID

JE049-5134

Conditions

Hereditary Angioedema (HAE)

Keywords

Hereditary angioedema

Study type

Observational

Recruitment status

Completed

Enrolment

1,761 participants (actual)

Lead sponsor

ShireIndustry (pharma, biotech, devices)

Collaborators

Takeda Development Center Americas, Inc.

Study start

10 Jul 2009 (actual)

Primary completion

31 May 2024 (actual)

Study completion

31 May 2024 (actual)

First posted

18 Dec 2009 (estimated)

Results

Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Observational model

Cohort

Time perspective

Prospective

Number of arms

1

Who may qualify

Sex

All

Healthy volunteers

Not accepted

Age groups

Child, Adult, Older adult

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

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.

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

• Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2024.
• Derived from this study: PubMed 35861129Maurer M, Aberer W, Caballero T, Bouillet L, Grumach AS, Botha J, Andresen I, Longhurst HJ; IOS Study Group. The Icatibant Outcome Survey: 10 years of experience with icatibant for patients with hereditary angioedema. Clin Exp Allergy. 2022 Sep;52(9):1048-1058. doi: 10.1111/cea.14206. Epub 2022 Aug 7.
• Derived from this study: PubMed 34965883Guilarte M, Sala-Cunill A, Baeza ML, Cabanas R, Hernandez MD, Ibanez E, de Larramendi CH, Lleonart R, Lobera T, Marques L, de San Pedro BS, Botha J, Andresen I, Caballero T; IOS Study Group. Hereditary angioedema due to C1 inhibitor deficiency: real-world experience from the Icatibant Outcome Survey in Spain. Allergy Asthma Clin Immunol. 2021 Dec 29;17(1):137. doi: 10.1186/s13223-021-00641-3.
• Derived from this study: PubMed 30127805Longhurst HJ, Dempster J, Lorenzo L, Buckland M, Grigoriadou S, Symons C, Bethune C, Fabien V, Bangs C, Garcez T. Real-world outcomes in hereditary angioedema: first experience from the Icatibant Outcome Survey in the United Kingdom. Allergy Asthma Clin Immunol. 2018 Aug 6;14:28. doi: 10.1186/s13223-018-0253-x. eCollection 2018.
• Derived from this study: PubMed 29599966Caballero T, Zanichelli A, Aberer W, Maurer M, Longhurst HJ, Bouillet L, Andresen I; IOS Study Group. Effectiveness of icatibant for treatment of hereditary angioedema attacks is not affected by body weight: findings from the Icatibant Outcome Survey, a cohort observational study. Clin Transl Allergy. 2018 Mar 23;8:11. doi: 10.1186/s13601-018-0195-x. eCollection 2018.
• Derived from this study: PubMed 28690642Aberer W, Maurer M, Bouillet L, Zanichelli A, Caballero T, Longhurst HJ, Perrin A, Andresen I; IOS Study Group. Breakthrough attacks in patients with hereditary angioedema receiving long-term prophylaxis are responsive to icatibant: findings from the Icatibant Outcome Survey. Allergy Asthma Clin Immunol. 2017 Jul 5;13:31. doi: 10.1186/s13223-017-0203-z. eCollection 2017.
• Derived from this study: PubMed 27742086Zanichelli A, Longhurst HJ, Maurer M, Bouillet L, Aberer W, Fabien V, Andresen I, Caballero T; IOS Study Group. Misdiagnosis trends in patients with hereditary angioedema from the real-world clinical setting. Ann Allergy Asthma Immunol. 2016 Oct;117(4):394-398. doi: 10.1016/j.anai.2016.08.014.
• Derived from this study: PubMed 27116379Longhurst HJ, Aberer W, Bouillet L, Caballero T, Maurer M, Fabien V, Zanichelli A; IOS Study Group. The Icatibant Outcome Survey: treatment of laryngeal angioedema attacks. Eur J Emerg Med. 2016 Jun;23(3):224-7. doi: 10.1097/MEJ.0000000000000292.
• Derived from this study: PubMed 26314822Longhurst HJ, Aberer W, Bouillet L, Caballero T, Fabien V, Zanichelli A, Maurer M; IOS Investigators. Analysis of characteristics associated with reinjection of icatibant: Results from the icatibant outcome survey. Allergy Asthma Proc. 2015 Sep-Oct;36(5):399-406. doi: 10.2500/aap.2015.36.3892.
• Derived from this study: PubMed 26112099Hernandez Fernandez de Rojas D, Ibanez E, Longhurst H, Maurer M, Fabien V, Aberer W, Bouillet L, Zanichelli A, Caballero T; IOS Study Group. Treatment of HAE Attacks in the Icatibant Outcome Survey: An Analysis of Icatibant Self-Administration versus Administration by Health Care Professionals. Int Arch Allergy Immunol. 2015;167(1):21-8. doi: 10.1159/000430864. Epub 2015 Jun 25.
• Derived from this study: PubMed 25198193Maurer M, Longhurst HJ, Fabien V, Li HH, Lumry WR. Treatment of hereditary angioedema with icatibant: efficacy in clinical trials versus effectiveness in the real-world setting. Allergy Asthma Proc. 2014 Sep-Oct;35(5):377-81. doi: 10.2500/aap.2014.35.3780. Epub 2014 Aug 6.
• To obtain more information on the study, click here/on this linkLink provided by the sponsor.
• Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

74 sites in 14 countries: see every site and its status.