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

NCT04957641 | Completed

• 2 other identifiers: TAK-743-4009MACS-2020-080401
• Study Type: observational
• Target: 221
• Locations: 20 countries
• Sites: 34

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.

Conditions

Hereditary Angioedema

Outcome Measures

Primary Outcomes (14)

• 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)

• 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)

• 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)

• 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)

• 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)

• 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)

• 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)

• 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)

• 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)

• 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)

• 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)

• 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)

• 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)

• 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 Outcomes (11)

• Healthcare Resource Utilization (HRU): Number of Visits to Healthcare Professionals (HCP)

  Throughout the study period (up to 6 months)

• HRU: Number of Participants With Emergency Room/Emergency Departments (ER/ED) Visits due to HAE Attacks

  Throughout the study period (up to 6 months)

• HRU: Number of Participants With Hospitalizations due to HAE Attacks

  Throughout the study period (up to 6 months)

• Health-related Quality of Life (HRQoL): Angioedema Quality of Life (AE-QoL) Questionnaire

  Throughout the study period (up to 6 months)

• HRQoL: EuroQoL Group 5-Dimension 5-Level (EQ-5D-5L) Questionnaire

  Throughout the study period (up to 6 months)

Study Arms (1)

Participants with HAE

Participants' data will be collected retrospectively from electronic medical records using chart review of de-identified data on participant demographics, HAE medical history and information on diagnostics, treatments and disease course assessments that are routinely performed in accordance with current guidelines and/or local standard of care which are entered into an Electronic data capture (EDC) system over multiple data collection waves up to 6 months.

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (36)

Medizinische Universitat Wien (Medical University of Vienna)

Vienna, 1090, Austria

Location

UZ Antwerpen

Edegem, 2650, Belgium

Location

University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

Sofia, 1431, Bulgaria

Location

Vancouver Allergy Clinic

Vancouver, British Columbia, BCV5Y1K2, Canada

Location

McMaster University Medical Centre

Hamilton, ON L8N 3Z5, Canada

Location

General Hospital Sibenik

Šibenik, 22000, Croatia

Location

Klinicki bolnicki centar Zagreb

Zagreb, 10000, Croatia

Location

Fakultni nemocnice Hradec Kralove

Hradec Králové, 500 05, Czechia

Location

Fakultni nemocnice v Motole

Prague, 150 06, Czechia

Location

East Tallinn Central Hospital

Tallinn, 10138, Estonia

Location

Universitätsklinikum Frankfurt

Frankfurt, 60590, Germany

Location

Navy Hospital of Athens

Athens, 115 21, Greece

Location

Laiko General Hospital of Athens

Athens, 115 27, Greece

Location

University General Hospital of Larissa

Larissa, 412 21, Greece

Location

Semmelweis Egyetem

Budapest, 1085, Hungary

Location

St James's Hospital

Dublin, D08 NHY1, Ireland

Location

Barzilai Medical Center

Ashkelon, Israel

Location

Bnai Zion Medical Center

Haifa, Israel

Location

Sheba Medical Center - PPDS

Ramat Gan, Israel

Location

Tel Aviv Sourasky Medical Center PPDS

Tel Aviv, Israel

Location

Pauls Stradins Clinical University Hospital

Riga, LV-1002, Latvia

Location

Centre of Investigation and Treatment of Allergic Diseases

Riga, LV-1003, Latvia

Location

Hospital of Lithuanian University of Health Sciences Kauno klinikos

Kaunas, 50161, Lithuania

Location

Vilnius University Hospital Santaros Klinikos

Vilnius, 08410, Lithuania

Location

Medical Faculty of Ss. Cyril and Methodius Univers

Skopje, 1000, North Macedonia

Location

Wojskowy Instytut Medyczny

Warsaw, 04-349, Poland

Location

Centro Hospitalar E Universitário de Coimbra EPE

Coimbra, 3004-561, Portugal

Location

Centro Hospitalar Lisboa Norte, E.P.E. - Hospital de Santa Maria

Lisbon, 1649-028, Portugal

Location

Centro Hospitalar de São João, E.P.E.

Porto, 4200-319, Portugal

Location

MediQuest Clinical Research Center

Sângeorgiu de Mures, 547530, Romania

Location

University Clinical Center of Serbia - PPDS

Belgrade, 11000, Serbia

Location

Univerzitna nemocnica Martin

Martin, 036 01, Slovakia

Location

Bolnisnica Golnik - Klinicni Oddelek za Pljucne Bolezni In Alergijo

Golnik, 4204, Slovenia

Location

Hospital Universitario Cruces

Barakaldo, 48903, Spain

Location

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, 08035, Spain

Location

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, 46026, Spain

Location

Related Publications (1)

• Farkas H, Aygoren-Pursun E, Ebo DG, Bara N, Psarros F, Gavini F, Bent-Ennakhil N, Sayegh L, Andresen I. Real-world treatment patterns and burden-of-disease of sub-optimally controlled hereditary angioedema. World Allergy Organ J. 2025 Sep 1;18(9):101100. doi: 10.1016/j.waojou.2025.101100. eCollection 2025 Sep.

  PMID: 40949786 DERIVED

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 5, 2021
• First Posted: July 12, 2021
• Study Start: April 21, 2022
• Primary Completion: January 13, 2023
• Study Completion: January 13, 2023
• Last Updated: June 26, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

NO (1); BU (1); CA (2); IE (1); RO (1); HU (1); IL (4); PL (1); SE (1); BE (1); DE (1); ES (3); AU (1); CZ (2); GR (3); LA (2); CR (2); SL (1); LI (2); PT (3)