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

Hereditary Angioedema (HAE) Clinical Trial

— PROSPECT  

Official title:

Treatment and Outcomes of Patients Identified With Non-Histaminergic Angioedema With Normal C1 Inhibitor in Canada

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05578417
• Other study ID #: TAK-743-4013
• Status: Recruiting
• Start date: June 15, 2023
• Est. completion date: June 30, 2024

Study information

• Verified date: April 2024
• Source: Takeda
• Contact: Takeda Contact
• Phone: +1-877-825-3327
• Email: medinfoUS[@]takeda.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: June 30, 2024
• Est. primary completion date: March 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Intervention

Other:
• No Intervention
As this is an observational study, no intervention will be administered.

Locations

Country	Name	City	State
Canada	Alberta Health Services	Calgary	Alberta
Canada	Alberta Health Services	Edmonton	Alberta
Canada	Hamilton Health Science Corporation	Hamilton	Ontario
Canada	CHU de Québec - Université Laval	Quebec
Canada	Vancouver Allergy Clinic	Vancouver	British Columbia
Canada	University of Manitoba	Winnipeg	Manitoba

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Country where clinical trial is conducted

Canada, 

Outcome

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

External Links

•   To obtain more information on the study, click here/on this link.