TAK-743-4009

Completed

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

Clinicaltrials.gov

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Other

#MACS-2020-080401

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.

IL

PT

GR

19+

Observational clinical trial.

What medical conditions were being studied?

Hereditary Angioedema (HAE)

How many participants were enrolled?

221

Were placebos part of the clinical trial?

No

When was the clinical trial conducted?

Apr 2022 - Jan 2023

How long was participation in the clinical trial?

Participants will stay in the study from providing consent to participate until they have completed the questionnaire (once only).

Sex

All

Age

12+ years

Healthy volunteers?

No

Boys, girls, men, and women can take part.

Must be teenagers of 12 years or older or adults at the time of their last documented hereditary angioedema (HAE) attack.

Must have been diagnosed with HAE type I or type II.

Must not have received Takhzyro as long-term prophylaxis since their diagnosis.

Additional entry criteria will be discussed with the study doctor.

Primary outcome measures

Number of Participants With HAE Attacks Occurrence During Study Period [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Categorized Based on Characteristics of HAE Attacks During Study Period [Time Frame: Throughout the study period (up to 6 months)]

Time Interval Between HAE Attacks During Study Period [Time Frame: Throughout the study period (up to 6 months)]

Duration of Each HAE Attacks During Study Period [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Based on Type of HAE Prophylactic Treatments [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Based on Long-term or Short-term HAE Prophylactic Treatment [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Initiation [Time Frame: Throughout the study period (up to 6 months)]

Duration of HAE Prophylactic Treatment [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Categorized Based on Dose and Route of Administration for HAE Prophylactic Treatment [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Discontinuation [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Based on Type of HAE On-demand Treatment [Time Frame: Throughout the study period (up to 6 months)]

Duration of HAE On-demand Treatment [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Categorized Based on Dose and Route of Administration for HAE On-demand Treatment [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Associated Between Prescriptions Patterns and HAE Attacks [Time Frame: Throughout the study period (up to 6 months)]

Secondary outcome measures

Healthcare Resource Utilization (HRU): Number of Visits to Healthcare Professionals (HCP) [Time Frame: Throughout the study period (up to 6 months)]

HRU: Number of Participants With Emergency Room/Emergency Departments (ER/ED) Visits due to HAE Attacks [Time Frame: Throughout the study period (up to 6 months)]

HRU: Number of Participants With Hospitalizations due to HAE Attacks [Time Frame: Throughout the study period (up to 6 months)]

Health-related Quality of Life (HRQoL): Angioedema Quality of Life (AE-QoL) Questionnaire [Time Frame: Throughout the study period (up to 6 months)]

HRQoL: EuroQoL Group 5-Dimension 5-Level (EQ-5D-5L) Questionnaire [Time Frame: Throughout the study period (up to 6 months)]

HRQoL: EuroQoL-five Dimensions, Youth Version (EQ-5D-Y) [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants With HAE Attacks Occurrence in Relevant Subgroups [Time Frame: Throughout the study period (up to 6 months)]

Number of Participants Categorized Based on Characteristics of HAE Attacks in Relevant Subgroups [Time Frame: Throughout the study period (up to 6 months)]

Time Interval Between HAE Attacks in Relevant Subgroups [Time Frame: Throughout the study period (up to 6 months)]

Duration of Each HAE Attacks in Relevant Subgroups [Time Frame: Throughout the study period (up to 6 months)]

HAE Attacks Prescription Pattern in Relevant Subgroups [Time Frame: Throughout the study period (up to 6 months)]

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

About this clinical trial

At a glance