Clinical trial NCT01034969: Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)
Official title: Icatibant Outcome Survey (IOS) Registry
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Angioedema (HAE)
Keywords: Hereditary angioedema
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda Development Center Americas, Inc.
First posted: 2009-12-18
Enrollment: 1761
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.

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 \[human\]).

Eligibility 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.

Primary outcomes:
measure: Incidence of Cardiac Ischemia Events in Participants Predisposed to Cardiac Ischemia Events With Concomitant Firazyr (Icatibant) Administration
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Hypotension for Firazyr (Icatibant)
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Swelling of Mucous Membranes for Firazyr (Icatibant)
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Bronchoconstriction for Firazyr (Icatibant)
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Aggravation of Pain for Firazyr (Icatibant)
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Sexual Hormones Level Measurements- Tanner Staging for Firazyr (Icatibant)
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Time to Complete Resolution of the Firazyr (Icatibant)-Treated Laryngeal Attacks
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Adverse Events (AE) Related to Firazyr (Icatibant)-Treated Laryngeal Attacks
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Adverse Drug Reactions (ADR) for Firazyr (Icatibant)
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Serious Adverse Events (SAE) for Firazyr (Icatibant)
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Pregnancy and Lactation Events During Firazyr (Icatibant) Exposure
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Adverse Events (AE) for Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Adverse Drug Reactions (ADR) for Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Serious Adverse Events (SAE) for Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Thrombotic or Thromboembolic Events for Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Incidence of Pregnancy and Lactation Events During Cinryze (C1 Inhibitor [Human]) Exposure
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Drug Exposure Data for Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Frequency of Hereditary Angioedema (HAE) Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Severity of Hereditary Angioedema Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Anatomic Location of Hereditary Angioedema Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Outcome of Severe or Laryngeal Hereditary Angioedema Attacks in Participants Treated With Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Outcome of Hereditary Angioedema Attacks for Treatment With Cinryze (C1 Inhibitor [Human])
timeFrame: From enrollment through study participation (Approximately 13 years)
Secondary outcomes:
measure: Time to Treatment For Attack
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Time to Complete Resolution of Attack
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Total Duration of Attack
timeFrame: From enrollment through study participation (Approximately 13 years)
measure: Hereditary Angioedema-Treated Attacks
timeFrame: From enrollment through study participation (Approximately 13 years)
Other outcomes:
Locations:
city: Campbelltown
country: Australia
facility: Campbelltown Hospital
state: New South Wales
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Graz
country: Austria
facility: Medizinische Universität Graz
city: Santo André
country: Brazil
facility: Faculdade de Medicina Do ABC
state: São Paulo
city: Brno
country: Czechia
facility: Fakultni nemocnice u sv. Anny v Brne
city: Odense
country: Denmark
facility: Odense Universitetshospital
city: Strasbourg
country: France
facility: Hopital de Hautepierre
state: Bas-Rhin
city: Caen
country: France
facility: Hopital Cote de Nacre
state: Calvados
city: Toulouse
country: France
facility: Hopital Purpan
state: Haute-Garonne
city: Angers Cedex 10
country: France
facility: CHU Angers
city: Bordeaux
country: France
facility: Centre Hospitalier Universitaire de Bordeaux, Hopital Pellegrin
city: Brest
country: France
facility: CHU La Cavale Blanche
city: Grenoble
country: France
facility: CHU de GRENOBLE
city: Le Mans cedex 9
country: France
facility: Centre Hospitalier Le Mans
city: Lille
country: France
facility: CHRU Lille
city: Lyon
country: France
facility: Groupement Hospitalier Edouard Herriot
city: Montpellier
country: France
facility: CHU Montpellier - Hôpital St Eloi
city: Nancy
country: France
facility: CHU de Nancy-Hopital Brabois Adulte
city: Nantes
country: France
facility: Hôtel Dieu - Nantes
city: Nice
country: France
facility: CHU de Nice Archet I
city: Niort
country: France
facility: Centre Hospitalier Georges Renon
city: Paris
country: France
facility: Hôtel Dieu de Paris Hospital
city: Paris
country: France
facility: Hôpital Saint Antoine
city: Paris
country: France
facility: Hopital Cochin
city: Reims
country: France
facility: CHU de Reims
city: Saint-Etienne
country: France
facility: Centre Hospitalier Universitaire de Saint Etienne
city: München
country: Germany
facility: Hals-Nasen-Ohrenklinik und Poliklinik
state: Bayern
city: Frankfurt
country: Germany
facility: Klinikum der Johann-Wolfgang Goethe-Universitat
state: Hessen
city: Essen
country: Germany
facility: Universitätsklinikum Essen
state: Nordrhein-Westfalen
city: Mainz
country: Germany
facility: Universitätsmedizin der Johannes Gutenberg-Universität Mainz
state: Rheinland-Pfalz
city: Dresden
country: Germany
facility: Universitätsklinikum Carl Gustav Carus an der TU Dresden
state: Sachsen
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Mörfelden-Walldorf
country: Germany
facility: Hämophilie Zentrum Rhein Main GmbH
city: Ulm
country: Germany
facility: Universitätsklinikum Ulm
city: Athens
country: Greece
facility: Navy Hospital of Athens
state: Attiki
city: Thessaloniki
country: Greece
facility: Papageorgiou General Hospital of Thessaloniki
city: Dublin 8
country: Ireland
facility: St James's Hospital
city: Haifa
country: Israel
facility: Bnai Zion Medical Center
city: Petach Tikva
country: Israel
facility: Rabin Medical Center - PPDS
city: Ramat-Gan
country: Israel
facility: Sheba Medical Center - PPDS
city: Tel Aviv
country: Israel
facility: Tel Aviv Sourasky Medical Center PPDS
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria Federico II
state: Campania
city: Milano
country: Italy
facility: ASST Fatebenefratelli Sacco - Ospedale Luigi Sacco
state: Lombardia
city: Bari
country: Italy
facility: AO Ospedale Policlinico Consorziale Di Bari
state: Puglia
city: Bari
country: Italy
facility: Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari
state: Puglia
city: Monserrato
country: Italy
facility: Presidio Policlinico Di Monserrato
state: Sardegna
city: Padova
country: Italy
facility: Universita degli Studi di Padova
city: Palermo
country: Italy
facility: Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello
city: Santiago de Compostela
country: Spain
facility: CHUS - H. Clinico U. de Santiago
state: A Coruña
city: Villajoyosa
country: Spain
facility: Hospital de La Marina Baixa
state: Alicante
city: L'Hospitalet de Llobregat
country: Spain
facility: Hospital Universitario de Bellvitge
state: Barcelona
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebrón - PPDS
city: Gijon
country: Spain
facility: Hospital Cruz Roja Española de Gijón
city: Jaen
country: Spain
facility: Hospital Universitario de Jaen
city: Lleida
country: Spain
facility: Hospital de Santa Maria
city: Logrono
country: Spain
facility: Centro de Alta Resolución de Procesos Asistenciales (C.A.R.P.A.)
city: Madrid
country: Spain
facility: Hospital General Universitario Gregorio Maranon
city: Madrid
country: Spain
facility: Hospital Clinico San Carlos
city: Madrid
country: Spain
facility: Hospital Universitario La Paz - PPDS
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio - PPDS
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia
city: Vigo
country: Spain
facility: Complejo Hospitalario Universitario de Vigo
city: Jönköping
country: Sweden
facility: Länssjukhuset Ryhov
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospitals NHS Foundation Trust
state: Cambridgeshire
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
state: Oxfordshire
city: Frimley
country: United Kingdom
facility: Frimley Park Hospital
state: Surrey
city: Leeds
country: United Kingdom
facility: St James University Hospital
state: York
city: Birmingham
country: United Kingdom
facility: Birmingham Heartlands Hospital
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales - PPDS
city: London
country: United Kingdom
facility: The Royal London Hospital
city: London
country: United Kingdom
facility: Royal Free Hospital
city: Manchester
country: United Kingdom
facility: Manchester Royal Infirmary - PPDS
city: Newcastle Upon Tyne
country: United Kingdom
facility: Royal Victoria Infirmary
city: Plymouth
country: United Kingdom
facility: Derriford Hospital
Study officials:
affiliation: Takeda Development Center Americas
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Maurer 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.
citation: Guilarte 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.
citation: Longhurst 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.
citation: Caballero 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.
citation: Aberer 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.
citation: Zanichelli 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.
citation: Longhurst 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.
citation: Longhurst 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.
citation: Hernandez 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.
citation: Maurer 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.