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

NCT05578417 | Completed

• 1 other identifier: TAK-743-4013
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 6

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.

Conditions

Hereditary Angioedema (HAE); Angioedema

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (19)

• 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

Study Arms (2)

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

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

Interventions

No Intervention OTHER

As this is an observational study, no intervention will be administered.

HAE nC1-INH; NHAE nC1-INH

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

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

You may qualify if:

• \. 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:
• Recurrent angioedema as documented by a healthcare professional within specialist charts
• Normal C4
• Normal C1 level and function
• Condition worsened with estrogen if estrogen is/was being received
• Lack of response to corticosteroid and high-dose regular and/or prophylactic antihistamine(s) treatment(s)
• Family history of non-histaminergic angioedema for patients with HAE nC1-INH

You may not qualify if:

• Other types of angioedema (type-1 HAE, type-2 HAE, acquired angioedema, etc.)
• Response to treatments used for histamine-related angioedema

Sponsors & Collaborators

• Takeda: lead

Study Sites (6)

Alberta Health Services

Calgary, Alberta, T2N 2T9, Canada

Location

Alberta Health Services

Edmonton, Alberta, T6G 2B7, Canada

Location

Vancouver Allergy Clinic

Vancouver, British Columbia, V5Z 1H8, Canada

Location

University of Manitoba

Winnipeg, Manitoba, R3A 1R9, Canada

Location

Hamilton Health Science Corporation

Hamilton, Ontario, L8S 4K1, Canada

Location

CHU de Québec - Université Laval

Québec, G1V 4G2, Canada

Location

Related Links

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

MeSH Terms

Conditions

Angioedemas, Hereditary; Angioedema

Condition Hierarchy (Ancestors)

Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 11, 2022
• First Posted: October 13, 2022
• Study Start: June 15, 2023
• Primary Completion: March 31, 2024
• Study Completion: June 30, 2024
• Last Updated: July 30, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

CA (6)