Clinical trial NCT04957641: A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults With Hereditary Angioedema
Official title: A Retrospective Observational Chart Review Study Evaluating the Burden of Illness and Treatment Patterns in Hereditary Angioedema Type I and II
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Angioedema
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2021-07-12
Enrollment: 221
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants aged greater than or equal to (\>=) 12 years with HAE type I or type II suffering from recurrent attacks will be enrolled in this study.

Brief summary: This study is about teenagers and adults with hereditary angioedema (HAE) type I and type II. In this study, the burden of illness means the impact of HAE in terms of long-term health outcomes and the financial cost.

The main aims of this study are as follows:

* to learn how often, how severe and where on the body HAE attacks occur.
* to describe how HAE prophylaxis and on-demand medicines are prescribed and used. (Prophylaxis medicines prevent a bleed from happening and on-demand medicines treat a bleed when it occurs.)

This study is about collecting data only; participants will not receive treatment as part of this study. Existing data available in the participant's medical records will be collected. Participants will be asked to complete an electronic questionnaire either on a website or by using an app on their mobile phone; data from this questionnaire will also be collected.

Participants do not need to visit their doctor in addition to their normal visits.


Eligibility criteria: Inclusion Criteria:

* Participant aged \>= 12 years at the time of the last documented HAE attack in the eligibility period.
* Physician-confirmed diagnosis (or confirmation in medical records) of HAE type I or type II.
* Participant had at least one documented HAE attack during the eligibility period.
* Participant is not adequately or sub-optimally controlled according to treating physician.
* Participant (or parent/legal guardian) is willing and able to comply with the study requirements and provides informed consent or assent.

Exclusion Criteria:

* Participant enrolled in a therapeutic investigational drug or device trial during the observation period.
* Participant initiated long-term prophylaxis with Takhzyro® at any time since diagnosis.

Primary outcomes:
measure: Number of Participants With HAE Attacks Occurrence During Study Period
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Categorized Based on Characteristics of HAE Attacks During Study Period
timeFrame: Throughout the study period (up to 6 months)
measure: Time Interval Between HAE Attacks During Study Period
timeFrame: Throughout the study period (up to 6 months)
measure: Duration of Each HAE Attacks During Study Period
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Based on Type of HAE Prophylactic Treatments
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Based on Long-term or Short-term HAE Prophylactic Treatment
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Initiation
timeFrame: Throughout the study period (up to 6 months)
measure: Duration of HAE Prophylactic Treatment
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Categorized Based on Dose and Route of Administration for HAE Prophylactic Treatment
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Discontinuation
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Based on Type of HAE On-demand Treatment
timeFrame: Throughout the study period (up to 6 months)
measure: Duration of HAE On-demand Treatment
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Categorized Based on Dose and Route of Administration for HAE On-demand Treatment
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Associated Between Prescriptions Patterns and HAE Attacks
timeFrame: Throughout the study period (up to 6 months)
Secondary outcomes:
measure: Healthcare Resource Utilization (HRU): Number of Visits to Healthcare Professionals (HCP)
timeFrame: Throughout the study period (up to 6 months)
measure: HRU: Number of Participants With Emergency Room/Emergency Departments (ER/ED) Visits due to HAE Attacks
timeFrame: Throughout the study period (up to 6 months)
measure: HRU: Number of Participants With Hospitalizations due to HAE Attacks
timeFrame: Throughout the study period (up to 6 months)
measure: Health-related Quality of Life (HRQoL): Angioedema Quality of Life (AE-QoL) Questionnaire
timeFrame: Throughout the study period (up to 6 months)
measure: HRQoL: EuroQoL Group 5-Dimension 5-Level (EQ-5D-5L) Questionnaire
timeFrame: Throughout the study period (up to 6 months)
measure: HRQoL: EuroQoL-five Dimensions, Youth Version (EQ-5D-Y)
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants With HAE Attacks Occurrence in Relevant Subgroups
timeFrame: Throughout the study period (up to 6 months)
measure: Number of Participants Categorized Based on Characteristics of HAE Attacks in Relevant Subgroups
timeFrame: Throughout the study period (up to 6 months)
measure: Time Interval Between HAE Attacks in Relevant Subgroups
timeFrame: Throughout the study period (up to 6 months)
measure: Duration of Each HAE Attacks in Relevant Subgroups
timeFrame: Throughout the study period (up to 6 months)
measure: HAE Attacks Prescription Pattern in Relevant Subgroups
timeFrame: Throughout the study period (up to 6 months)
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Medizinische Universitat Wien (Medical University of Vienna)
city: Edegem
country: Belgium
facility: UZ Antwerpen
city: Sofia
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
city: Vancouver
country: Canada
facility: Vancouver Allergy Clinic
state: British Columbia
city: Hamilton
country: Canada
facility: McMaster University Medical Centre
city: Zagreb
country: Croatia
facility: Klinicki bolnicki centar Zagreb
city: Šibenik
country: Croatia
facility: General Hospital Sibenik
city: Hradec Králové
country: Czechia
facility: Fakultni nemocnice Hradec Kralove
city: Praha
country: Czechia
facility: Fakultni nemocnice v Motole
city: Tallinn
country: Estonia
facility: East Tallinn Central Hospital
city: Frankfurt
country: Germany
facility: Universitätsklinikum Frankfurt
city: Athens
country: Greece
facility: Navy Hospital of Athens
city: Athens
country: Greece
facility: Laiko General Hospital of Athens
city: Larisa
country: Greece
facility: University General Hospital of Larissa
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Dublin
country: Ireland
facility: St James's Hospital
city: Ashkelon
country: Israel
facility: Barzilai Medical Center
city: Haifa
country: Israel
facility: Bnai Zion Medical Center
city: Ramat Gan
country: Israel
facility: Sheba Medical Center - PPDS
city: Tel Aviv-Yafo
country: Israel
facility: Tel Aviv Sourasky Medical Center PPDS
city: Rīga
country: Latvia
facility: Pauls Stradins Clinical University Hospital
city: Rīga
country: Latvia
facility: Centre of Investigation and Treatment of Allergic Diseases
city: Kaunas
country: Lithuania
facility: Hospital of Lithuanian University of Health Sciences Kauno klinikos
city: Vilnius
country: Lithuania
facility: Vilnius University Hospital Santaros Klinikos
city: Skopje
country: North Macedonia
facility: Medical Faculty of Ss. Cyril and Methodius Univers
city: Warszawa
country: Poland
facility: Wojskowy Instytut Medyczny
city: Coimbra
country: Portugal
facility: Centro Hospitalar E Universitário de Coimbra EPE
city: Lisboa
country: Portugal
facility: Centro Hospitalar Lisboa Norte, E.P.E. - Hospital de Santa Maria
city: Porto
country: Portugal
facility: Centro Hospitalar de São João, E.P.E.
city: Sângeorgiu de Mures
country: Romania
facility: MediQuest Clinical Research Center
city: Belgrade
country: Serbia
facility: University Clinical Center of Serbia - PPDS
city: Martin
country: Slovakia
facility: Univerzitna nemocnica Martin
city: Golnik
country: Slovenia
facility: Bolnisnica Golnik - Klinicni Oddelek za Pljucne Bolezni In Alergijo
city: Barakaldo
country: Spain
facility: Hospital Universitario Cruces
city: Barcelona
country: Spain
facility: Hospital Universitario Vall d'Hebron - PPDS
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: