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

Completed

• Conditions: Hereditary Angioedema (HAE); Angioedema
• Interventions: Other: No Intervention

• Registration Number: NCT05578417
• Lead Sponsor: Takeda

Brief Summary

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.

Detailed 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.

Study Timeline

• Study First Submitted: 2022-10-11
• Study First Submitted QC: 2022-10-11
• Study First Posted: 2022-10-13
• Study Start: 2023-06-15
• Primary Completion: 2024-03-31
• Study Completion: 2024-06-30
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-29
• Last Update Posted: 2024-07-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 60

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

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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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
NHAE nC1-INH	No Intervention	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.
HAE nC1-INH	No Intervention	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants Categorized by Type of Treatments Received by Participants	Up to approximately 10 years
Number of Walk-in Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH	Up to approximately 10 years
Number of Participants Receiving Multiple Long-Term Prophylaxis (LTP) Therapies at Once	Up to approximately 10 years	Number of participants will be reported by number of LTP therapies received at once.
Number of Participants Receiving Multiple LTP Therapies Categorized by Combination and Order of Treatments Received	Up to approximately 10 years
Number of Attacks Occurring per Three-Months	Before and after initiation of treatment up to end of the study (up to approximately 10 years)
Number of Participants Diagnosed With HAE nC1-INH at Each Site	Up to approximately 10 years
Number of Participants by Body Sites Affected by Attacks per Three Months	Before and after initiation of treatment up to end of the study (up to approximately 10 years)	Body sites will include face, lip, tongue, gastrointestinal (GI) system, larynx, extremities, and other.
Number of Participants with Symptoms of the Attack	Up to approximately 10 years
Time from Treatment (On-demand Therapy) to Symptom Improvement/Resolution	Up to approximately 10 years
Number of Emergency Room Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH	Up to approximately 10 years
Number of Participants Diagnosed With NHAE nC1-INH at Each Site	Up to approximately 10 years
Average Duration of Treatment for Each Type of Treatment Received by Participants	Up to approximately 10 years
Number of Participants Categorized by Type of Settings Where Treatment was Received	Up to approximately 10 years	Categories of treatment settings include: home, hospital, physician office, emergency room visits (ER), and other.
Average Dose for Each Type of Treatment Received by Participants	Up to approximately 10 years
Number of Participants Categorized by Severity of Attack per Three Months	Before and after initiation of treatment up to end of the study (up to approximately 10 years)	Categories of severity are planned to include ER, use of rescue medication(s), (%) intubation, hospitalization, and laryngeal involvement.
Number of Primary Care Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH	Up to approximately 10 years
Number of Unscheduled Physician Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH	Up to approximately 10 years
Number of Hospitalizations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH	Up to approximately 10 years
Number of Intubations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH	Up to approximately 10 years

Trial Locations

Locations (5)

CHU de Québec - Université Laval; 🇨🇦 Quebec, Canada

Hamilton Health Science Corporation; 🇨🇦 Hamilton, Ontario, Canada

University of Manitoba; 🇨🇦 Winnipeg, Manitoba, Canada

Alberta Health Services; 🇨🇦 Edmonton, Alberta, Canada

Vancouver Allergy Clinic; 🇨🇦 Vancouver, British Columbia, Canada