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

Takeda

Status: Completed

Conditions: Hereditary Angioedema

Study type: Observational

Funder types: Industry

Identifiers

NCT04957641

TAK-743-4009

MACS-2020-080401 (Other Identifier)

Details and patient eligibility

About

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.

Enrollment: 221 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

221 participants in 1 patient group

Participants with HAE

Description:

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.