Firazyr® Patient Registry (Icatibant Outcome Survey - IOS)

Hereditary Angioedema (HAE) Clinical Trial

Official title:

Icatibant Outcome Survey (IOS) Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01034969
• Other study ID #: JE049-5134
• Status: Recruiting
• Start date: July 10, 2009
• Est. completion date: January 31, 2027

Study information

• Verified date: September 2023
• Source: Takeda
• Contact: Takeda Development Center Americas Contact
• Phone: +1 866 842 5335
• Email: ClinicalTransparency[@]takeda.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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]).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 3000
• Est. completion date: January 31, 2027
• Est. primary completion date: January 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Locations

Country	Name	City	State
Australia	Royal Adelaide Hospital	Adelaide	South Australia
Australia	Campbelltown Hospital	Campbelltown	New South Wales
Austria	Medizinische Universität Graz	Graz
Brazil	Faculdade de Medicina Do ABC	Santo André	São Paulo
Czechia	Fakultni nemocnice u sv. Anny v Brne	Brno
Denmark	Odense Universitetshospital	Odense
France	CHU Angers	Angers Cedex 10
France	Centre Hospitalier Universitaire de Bordeaux, Hopital Pellegrin	Bordeaux
France	CHU La Cavale Blanche	Brest
France	Hopital Cote de Nacre	Caen	Calvados
France	CHU de GRENOBLE	Grenoble
France	Centre Hospitalier Le Mans	Le Mans cedex 9
France	CHRU Lille	Lille
France	Groupement Hospitalier Edouard Herriot	Lyon
France	CHU Montpellier - Hôpital St Eloi	Montpellier
France	CHU de Nancy-Hopital Brabois Adulte	Nancy
France	Hôtel Dieu - Nantes	Nantes
France	CHU de Nice Archet I	Nice
France	Centre Hospitalier Georges Renon	Niort
France	Hopital Cochin	Paris
France	Hôpital Saint Antoine	Paris
France	Hôtel Dieu de Paris Hospital	Paris
France	CHU de Reims	Reims
France	Centre Hospitalier Universitaire de Saint Etienne	Saint-Etienne
France	Hopital de Hautepierre	Strasbourg	Bas-Rhin
France	Hopital Purpan	Toulouse	Haute-Garonne
Germany	Charité - Universitätsmedizin Berlin	Berlin
Germany	Universitätsklinikum Carl Gustav Carus an der TU Dresden	Dresden	Sachsen
Germany	Universitätsklinikum Essen	Essen	Nordrhein-Westfalen
Germany	Klinikum der Johann-Wolfgang Goethe-Universitat	Frankfurt	Hessen
Germany	Universitätsmedizin der Johannes Gutenberg-Universität Mainz	Mainz	Rheinland-Pfalz
Germany	Hämophilie Zentrum Rhein Main GmbH	Mörfelden-Walldorf
Germany	Hals-Nasen-Ohrenklinik und Poliklinik	München	Bayern
Germany	Universitätsklinikum Ulm	Ulm
Greece	Navy Hospital of Athens	Athens	Attiki
Greece	Papageorgiou General Hospital of Thessaloniki	Thessaloniki
Ireland	St James's Hospital	Dublin 8
Israel	Bnai Zion Medical Center	Haifa
Israel	Rabin Medical Center - PPDS	Petach Tikva
Israel	Sheba Medical Center - PPDS	Ramat-Gan
Israel	Tel Aviv Sourasky Medical Center PPDS	Tel Aviv
Italy	AO Ospedale Policlinico Consorziale Di Bari	Bari	Puglia
Italy	Azienda Ospedaliero Universitaria Consorziale Policlinico di Bari	Bari	Puglia
Italy	ASST Fatebenefratelli Sacco - Ospedale Luigi Sacco	Milano	Lombardia
Italy	Presidio Policlinico Di Monserrato	Monserrato	Sardegna
Italy	Azienda Ospedaliera Universitaria Federico II	Napoli	Campania
Italy	Universita degli Studi di Padova	Padova
Italy	Azienda Ospedaliera Ospedali Riuniti Villa Sofia-Cervello	Palermo
Spain	Hospital Universitario Vall d'Hebrón - PPDS	Barcelona
Spain	Hospital Cruz Roja Española de Gijón	Gijon
Spain	Hospital Universitario de Jaen	Jaen
Spain	Hospital Universitario de Bellvitge	L'Hospitalet de Llobregat	Barcelona
Spain	Hospital de Santa Maria	Lleida
Spain	Centro de Alta Resolución de Procesos Asistenciales (C.A.R.P.A.)	Logrono
Spain	Hospital Clinico San Carlos	Madrid
Spain	Hospital General Universitario Gregorio Maranon	Madrid
Spain	Hospital Universitario La Paz - PPDS	Madrid
Spain	CHUS - H. Clinico U. de Santiago	Santiago de Compostela	A Coruña
Spain	Hospital Universitario Virgen del Rocio - PPDS	Sevilla
Spain	Hospital Universitari i Politecnic La Fe de Valencia	Valencia
Spain	Complejo Hospitalario Universitario de Vigo	Vigo
Spain	Hospital de La Marina Baixa	Villajoyosa	Alicante
Sweden	Länssjukhuset Ryhov	Jönköping
United Kingdom	Birmingham Heartlands Hospital	Birmingham
United Kingdom	Cambridge University Hospitals NHS Foundation Trust	Cambridge	Cambridgeshire
United Kingdom	University Hospital of Wales - PPDS	Cardiff
United Kingdom	Frimley Park Hospital	Frimley	Surrey
United Kingdom	St James University Hospital	Leeds	York
United Kingdom	Royal Free Hospital	London
United Kingdom	The Royal London Hospital	London
United Kingdom	Manchester Royal Infirmary - PPDS	Manchester
United Kingdom	Royal Victoria Infirmary	Newcastle Upon Tyne
United Kingdom	John Radcliffe Hospital	Oxford	Oxfordshire
United Kingdom	Derriford Hospital	Plymouth

Sponsors (2)

Lead Sponsor	Collaborator
Shire	Takeda Development Center Americas, Inc.

Countries where clinical trial is conducted

Australia,  Austria,  Brazil,  Czechia,  Denmark,  France,  Germany,  Greece,  Ireland,  Israel,  Italy,  Spain,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	Incidence of Hypotension for Firazyr (Icatibant)	Incidence of hypotension for Firazyr (Icatibant) will be assessed.	From enrollment through study participation (Approximately 13 years)
Primary	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)
Primary	Incidence of Bronchoconstriction for Firazyr (Icatibant)	Incidence of bronchoconstriction for Firazyr (Icatibant) will be assessed.	From enrollment through study participation (Approximately 13 years)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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	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)
Secondary	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)
Secondary	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)
Secondary	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)

External Links

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