A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults With Hereditary Angioedema

Hereditary Angioedema Clinical Trial

— BOISTERN  

Official title:

A Retrospective Observational Chart Review Study Evaluating the Burden of Illness and Treatment Patterns in Hereditary Angioedema Type I and II

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04957641
• Other study ID #: TAK-743-4009
• Secondary ID: MACS-2020-080401
• Status: Completed
• Start date: April 21, 2022
• Est. completion date: January 13, 2023

Study information

• Verified date: June 2023
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 221
• Est. completion date: January 13, 2023
• Est. primary completion date: January 13, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema

Locations

Country	Name	City	State
Austria	Medizinische Universitat Wien (Medical University of Vienna)	Vienna
Belgium	UZ Antwerpen	Edegem
Bulgaria	University Multiprofile Hospital for Active Treatment Aleksandrovska EAD	Sofia
Canada	McMaster University Medical Centre	Hamilton
Canada	Vancouver Allergy Clinic	Vancouver	British Columbia
Croatia	General Hospital Sibenik	Šibenik
Croatia	Klinicki bolnicki centar Zagreb	Zagreb
Czechia	Fakultni nemocnice Hradec Kralove	Hradec Králové
Czechia	Fakultni nemocnice v Motole	Praha
Estonia	East Tallinn Central Hospital	Tallinn
Germany	Universitätsklinikum Frankfurt	Frankfurt
Greece	Laiko General Hospital of Athens	Athens
Greece	Navy Hospital of Athens	Athens
Greece	University General Hospital of Larissa	Larisa
Hungary	Semmelweis Egyetem	Budapest
Ireland	St James's Hospital	Dublin
Israel	Barzilai Medical Center	Ashkelon
Israel	Bnai Zion Medical Center	Haifa
Israel	Sheba Medical Center - PPDS	Ramat Gan
Israel	Tel Aviv Sourasky Medical Center PPDS	Tel Aviv-Yafo
Latvia	Centre of Investigation and Treatment of Allergic Diseases	Riga
Latvia	Pauls Stradins Clinical University Hospital	Riga
Lithuania	Hospital of Lithuanian University of Health Sciences Kauno klinikos	Kaunas
Lithuania	Vilnius University Hospital Santaros Klinikos	Vilnius
North Macedonia	Medical Faculty of Ss. Cyril and Methodius Univers	Skopje
Poland	Wojskowy Instytut Medyczny	Warszawa
Portugal	Centro Hospitalar E Universitário de Coimbra EPE	Coimbra
Portugal	Centro Hospitalar Lisboa Norte, E.P.E. - Hospital de Santa Maria	Lisboa
Portugal	Centro Hospitalar de São João, E.P.E.	Porto
Romania	MediQuest Clinical Research Center	Sângeorgiu de Mures
Serbia	University Clinical Center of Serbia - PPDS	Belgrade
Slovakia	Univerzitna nemocnica Martin	Martin
Slovenia	Bolnisnica Golnik - Klinicni Oddelek za Pljucne Bolezni In Alergijo	Golnik
Spain	Hospital Universitario Cruces	Barakaldo
Spain	Hospital Universitario Vall d'Hebron - PPDS	Barcelona
Spain	Hospital Universitari i Politecnic La Fe de Valencia	Valencia

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Countries where clinical trial is conducted

Austria,  Belgium,  Bulgaria,  Canada,  Croatia,  Czechia,  Estonia,  Germany,  Greece,  Hungary,  Ireland,  Israel,  Latvia,  Lithuania,  North Macedonia,  Poland,  Portugal,  Romania,  Serbia,  Slovakia,  Slovenia,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With HAE Attacks Occurrence During Study Period	HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Number of participants with HAE attacks occurrence (start and end date of each HAE attack) during study period will be reported.	Throughout the study period (up to 6 months)
Primary	Number of Participants Categorized Based on Characteristics of HAE Attacks During Study Period	HAE attack characteristics will be based on prodromal symptoms, severity and location, whether attack was life-threatening will be reported.	Throughout the study period (up to 6 months)
Primary	Time Interval Between HAE Attacks During Study Period	Time interval between HAE attacks during the study period will be reported.	Throughout the study period (up to 6 months)
Primary	Duration of Each HAE Attacks During Study Period	Duration of each HAE attacks during study period will be reported.	Throughout the study period (up to 6 months)
Primary	Number of Participants Based on Type of HAE Prophylactic Treatments	Number of participants based on type of HAE prophylactic treatment will be reported.	Throughout the study period (up to 6 months)
Primary	Number of Participants Based on Long-term or Short-term HAE Prophylactic Treatment	Number of participants based on long-term or short-term HAE prophylactic treatment will be reported.	Throughout the study period (up to 6 months)
Primary	Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Initiation	Number of participants categorized based on reasons for HAE prophylactic treatment initiation will be reported.	Throughout the study period (up to 6 months)
Primary	Duration of HAE Prophylactic Treatment	Duration of HAE prophylactic treatment includes treatment start and end dates will be reported.	Throughout the study period (up to 6 months)
Primary	Number of Participants Categorized Based on Dose and Route of Administration for HAE Prophylactic Treatment	Number of participants categorized based on dose and route of administration for HAE prophylactic treatment will be reported.	Throughout the study period (up to 6 months)
Primary	Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Discontinuation	Number of participants categorized based on reasons for HAE prophylactic treatment discontinuation will be reported.	Throughout the study period (up to 6 months)
Primary	Number of Participants Based on Type of HAE On-demand Treatment	Number of participants based on type of HAE on-demand treatment will be reported.	Throughout the study period (up to 6 months)
Primary	Duration of HAE On-demand Treatment	Duration of HAE on-demand treatment includes treatment start and end dates will be reported.	Throughout the study period (up to 6 months)
Primary	Number of Participants Categorized Based on Dose and Route of Administration for HAE On-demand Treatment	Number of participants categorized based on dose, route of administration and setting of administration for HAE on-demand treatment will be reported.	Throughout the study period (up to 6 months)
Primary	Number of Participants Associated Between Prescriptions Patterns and HAE Attacks	Number of participants with association between prescriptions patterns and HAE attacks related to frequency or severity. Generalized linear models (GLM) will be used to evaluate the association of outcomes of interest with relevant covariates between prescription pattern, HAE attacks, frequency and severity of HAE attacks will be reported.	Throughout the study period (up to 6 months)
Secondary	Healthcare Resource Utilization (HRU): Number of Visits to Healthcare Professionals (HCP)	HRU is measured by the number of visits to HCP will be reported.	Throughout the study period (up to 6 months)
Secondary	HRU: Number of Participants With Emergency Room/Emergency Departments (ER/ED) Visits due to HAE Attacks	Number of participants with ER/ED visits due to HAE attacks will be reported.	Throughout the study period (up to 6 months)
Secondary	HRU: Number of Participants With Hospitalizations due to HAE Attacks	Number of participants with hospitalizations due to HAE attacks will be reported.	Throughout the study period (up to 6 months)
Secondary	Health-related Quality of Life (HRQoL): Angioedema Quality of Life (AE-QoL) Questionnaire	The AE-QoL was developed to measure HRQoL in participants with recurrent angioedema. It is a self-administered patient-reported outcome instrument with a recall period of four weeks. There are 17 items across four domains: functioning (four items), fatigue/mood (five items), fears/shame (six items), and food (two items). Responses use a 5-point Likert scale ranging from 1 (Never) to 5 (Very Often). Global scores range from 0 to 100 and scores by domains range from 0 to 100. An overall score is calculated and a higher score indicates lower quality of life.	Throughout the study period (up to 6 months)
Secondary	HRQoL: EuroQoL Group 5-Dimension 5-Level (EQ-5D-5L) Questionnaire	The EQ-5D-5L descriptive system assesses health in five dimensions (Mobility, Self-Care, Usual Activities, Pain / Discomfort, Anxiety / Depression), each of which has five levels of response (no problems, slight problems, moderate problems, severe problems, extreme problems/unable to). This part of the EQ-5D questionnaire provides a descriptive profile that can be used to generate a health state profile. Health state index scores generally range from less than 0 (where 0 is the value of a health state equivalent to dead; negative values representing values as worse than dead) to 1 (the value of full health), with higher scores indicating higher health utility. The second part of the questionnaire consists of a visual analogue scale (VAS) on which the participant rates his/her perceived health from 0 (the worst imaginable health) to 100 (the best imaginable health).	Throughout the study period (up to 6 months)
Secondary	HRQoL: EuroQoL-five Dimensions, Youth Version (EQ-5D-Y)	The EQ-5D-Y is composed of two sections. The first section contains one question for each of the five dimensions: mobility, looking after myself, doing usual activities, having pain or discomfort, and feeling worried, sad, or unhappy. Participants select from three response levels (no problems, some problems, a lot of problems) for each dimension. A health state profile score can be calculated from the responses on these five questions. The second section includes the same visual analogue scale as the EQ-5D-5L. The scale is scored from 0-100. The reference to a high score indicates a better outcome of quality of life.	Throughout the study period (up to 6 months)
Secondary	Number of Participants With HAE Attacks Occurrence in Relevant Subgroups	HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE). Number of participants with HAE attacks occurrence (start and end date of each HAE attack) in relevant subgroups will be reported.	Throughout the study period (up to 6 months)
Secondary	Number of Participants Categorized Based on Characteristics of HAE Attacks in Relevant Subgroups	HAE attack characteristics will be based on prodromal symptoms, severity and location, whether attack was life-threatening in relevant subgroups will be reported. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).	Throughout the study period (up to 6 months)
Secondary	Time Interval Between HAE Attacks in Relevant Subgroups	Time interval between HAE attacks in relevant subgroups will be reported. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).	Throughout the study period (up to 6 months)
Secondary	Duration of Each HAE Attacks in Relevant Subgroups	Duration of each HAE attacks in relevant subgroups will be reported. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).	Throughout the study period (up to 6 months)
Secondary	HAE Attacks Prescription Pattern in Relevant Subgroups	Prescription pattern of HAE attacks medication by drug class in relevant participant subgroups during study period. Relevant subgroups is defined by disease characteristics (e.g., number of attacks) and treatment patterns (type of treatments for HAE).	Throughout the study period (up to 6 months)

External Links

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