A Study to Review the Treatment and Outcomes of Teenagers and Adults With Non-histaminergic Angioedema With Normal C1 Inhibitor in Canada (PROSPECT)

Takeda

Status: Completed

Conditions: Angioedema; Hereditary Angioedema (HAE)

Treatments: Other: No Intervention

Study type: Observational

Funder types: Industry

Identifiers

NCT05578417

TAK-743-4013

Details and patient eligibility

About

The main aim of this study is to describe the characteristics of participants, check for number of hereditary angioedema (HAE) cases, their treatment and outcomes. Another aim is to check how the healthcare facilities were utilized for treatment.

Participants' data will be taken from their medical records (charts), which were already collected as a part of their routine care between January 1, 2012, and January 1, 2022.

Full description

This is a non-interventional, retrospective, observational study of participants with Hereditary Angioedema with normal C1 esterase inhibitor (HAE) and Non-histaminergic Angioedema (NHAE) with normal C1-inhibitor function (nC1-INH) receiving various treatments between January 1, 2012, and January 1, 2022. The study will assess case numbers, treatments, outcomes, and healthcare resource utilization in the real-world setting.

This study will enroll approximately 90 to 150 participants. Participants will be enrolled in the following two cohorts:

• Participants with HAE nC1-INH
• Participants with NHAE nC1-INH

This study will have a retrospective data collection from January 1, 2012 to January 1, 2022 by using data from the participant medical charts that were already collected as part of routine care.

This multi-center trial will be conducted in Canada. The overall time for data collection in this study will be approximately 10 years.

Enrollment: 60 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Participants ≥12 years of age with HAE nC1-INH or NHAE nC1-INH diagnosed by an HAE treating specialist based on some or all of the following:

2. Recurrent angioedema as documented by a healthcare professional within specialist charts

3. Normal C4

4. Normal C1 level and function

5. Condition worsened with estrogen if estrogen is/was being received

6. Lack of response to corticosteroid and high-dose regular and/or prophylactic antihistamine(s) treatment(s)

7. Family history of non-histaminergic angioedema for patients with HAE nC1-INH

Exclusion criteria

1. Other types of angioedema (type-1 HAE, type-2 HAE, acquired angioedema, etc.)
2. Not meeting the above diagnostic criteria for inclusion
3. Response to treatments used for histamine-related angioedema

Trial design

60 participants in 2 patient groups

HAE nC1-INH

Description:

Participants with HAE nC1-INH will be observed retrospectively for the treatments they receive and their outcomes in the real-world setting from January 1, 2012, to January 1, 2022.

Treatment:

Other: No Intervention

NHAE nC1-INH

Description:

Participants with NHAE nC1-INH will be observed retrospectively for the treatments they receive and their outcomes in the real-world setting from January 1, 2012, to January 1, 2022.

Treatment:

Other: No Intervention