Clinical trial NCT05578417: A Study to Review the Treatment and Outcomes of Teenagers and Adults With Non-histaminergic Angioedema With Normal C1 Inhibitor in Canada
Official title: Treatment and Outcomes of Patients Identified With Non-Histaminergic Angioedema With Normal C1 Inhibitor in Canada
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Angioedema (HAE)
Angioedema
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2022-10-13
Enrollment: 60
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants with confirmed diagnosis of HAE nC1-INH or NHAE nC1-INH prior to January 1, 2022.

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.

Eligibility criteria: 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:

1. Recurrent angioedema as documented by a healthcare professional within specialist charts
2. Normal C4
3. Normal C1 level and function
4. Condition worsened with estrogen if estrogen is/was being received
5. Lack of response to corticosteroid and high-dose regular and/or prophylactic antihistamine(s) treatment(s)
6. 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

Primary outcomes:
measure: Number of Participants Diagnosed With HAE nC1-INH at Each Site
timeFrame: Up to approximately 10 years
measure: Number of Participants Diagnosed With NHAE nC1-INH at Each Site
timeFrame: Up to approximately 10 years
measure: Number of Participants Categorized by Type of Treatments Received by Participants
timeFrame: Up to approximately 10 years
measure: Average Dose for Each Type of Treatment Received by Participants
timeFrame: Up to approximately 10 years
measure: Average Duration of Treatment for Each Type of Treatment Received by Participants
timeFrame: Up to approximately 10 years
measure: Number of Participants Categorized by Type of Settings Where Treatment was Received
timeFrame: Up to approximately 10 years
measure: Number of Participants Receiving Multiple Long-Term Prophylaxis (LTP) Therapies at Once
timeFrame: Up to approximately 10 years
measure: Number of Participants Receiving Multiple LTP Therapies Categorized by Combination and Order of Treatments Received
timeFrame: Up to approximately 10 years
measure: Number of Attacks Occurring per Three-Months
timeFrame: Before and after initiation of treatment up to end of the study (up to approximately 10 years)
measure: Number of Participants Categorized by Severity of Attack per Three Months
timeFrame: Before and after initiation of treatment up to end of the study (up to approximately 10 years)
measure: Number of Participants by Body Sites Affected by Attacks per Three Months
timeFrame: Before and after initiation of treatment up to end of the study (up to approximately 10 years)
measure: Number of Participants with Symptoms of the Attack
timeFrame: Up to approximately 10 years
measure: Time from Treatment (On-demand Therapy) to Symptom Improvement/Resolution
timeFrame: Up to approximately 10 years
measure: Number of Primary Care Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
timeFrame: Up to approximately 10 years
measure: Number of Walk-in Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
timeFrame: Up to approximately 10 years
measure: Number of Unscheduled Physician Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
timeFrame: Up to approximately 10 years
measure: Number of Emergency Room Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
timeFrame: Up to approximately 10 years
measure: Number of Hospitalizations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
timeFrame: Up to approximately 10 years
measure: Number of Intubations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
timeFrame: Up to approximately 10 years
Secondary outcomes:
Other outcomes:
Locations:
city: Calgary
country: Canada
facility: Alberta Health Services
state: Alberta
city: Edmonton
country: Canada
facility: Alberta Health Services
state: Alberta
city: Vancouver
country: Canada
facility: Vancouver Allergy Clinic
state: British Columbia
city: Winnipeg
country: Canada
facility: University of Manitoba
state: Manitoba
city: Hamilton
country: Canada
facility: Hamilton Health Science Corporation
state: Ontario
city: Quebec
country: Canada
facility: CHU de Québec - Université Laval
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: