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

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

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.

Study Overview

• Status: Completed
• Conditions: Angioedema; Hereditary Angioedema (HAE)
• Intervention / Treatment: Other: No Intervention

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 Type: Observational
• Enrollment (Actual): 60

Contacts and Locations

Study Locations

• Canada
  • Quebec, Canada, G1V 4G2
    • CHU de Quebec - Universite Laval
  • Alberta
    • Calgary, Alberta, Canada, T2N 2T9
      • Alberta Health services
    • Edmonton, Alberta, Canada, T6G 2B7
      • Alberta Health services
  • British Columbia
    • Vancouver, British Columbia, Canada, V5Z 1H8
      • Vancouver Allergy Clinic
  • Manitoba
    • Winnipeg, Manitoba, Canada, R3A 1R9
      • University of Manitoba
  • Ontario
    • Hamilton, Ontario, Canada, L8S 4K1
      • Hamilton Health Science Corporation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Participants with confirmed diagnosis of HAE nC1-INH or NHAE nC1-INH prior to January 1, 2022.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
HAE nC1-INH; 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.	Other: No Intervention; As this is an observational study, no intervention will be administered.
NHAE nC1-INH; 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.	Other: No Intervention; As this is an observational study, no intervention will be administered.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Diagnosed With HAE nC1-INH at Each Site		Up to approximately 10 years
Number of Participants Diagnosed With NHAE nC1-INH at Each Site		Up to approximately 10 years
Number of Participants Categorized by Type of Treatments Received by Participants		Up to approximately 10 years
Average Dose for Each Type of Treatment Received by Participants		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	Categories of treatment settings include: home, hospital, physician office, emergency room visits (ER), and other.	Up to approximately 10 years
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
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 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)
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)
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 Primary Care Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH		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 Unscheduled Physician Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH		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 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

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

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

Study record dates

Study Major Dates

• Study Start (Actual): June 15, 2023
• Primary Completion (Actual): March 31, 2024
• Study Completion (Actual): June 30, 2024

Study Registration Dates

• First Submitted: October 11, 2022
• First Submitted That Met QC Criteria: October 11, 2022
• First Posted (Actual): October 13, 2022

Study Record Updates

• Last Update Posted (Actual): July 30, 2024
• Last Update Submitted That Met QC Criteria: July 29, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: Drug Therapy
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Skin Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Hypersensitivity, Immediate; Genetic Diseases, Inborn; Skin Diseases, Vascular; Hypersensitivity; Urticaria; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Angioedema; Angioedemas, Hereditary
• Other Study ID Numbers: TAK-743-4013

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No