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Completed

NCT Number: NCT05578417

A Study to Review the Treatment and Outcomes of Teenagers and Adults 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.

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Key information

Conditions

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

Age range

12 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Alberta Health Services, Calgary, Alberta, Canada

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • Other types of angioedema (type-1 HAE, type-2 HAE, acquired angioedema, etc.)
  • Not meeting the above diagnostic criteria for inclusion
  • Response to treatments used for histamine-related angioedema

Treatment and study plan

No intervention

Other

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

Primary outcomes

  1. Number of Participants Diagnosed With HAE nC1-INH at Each Site

    Time frame: Up to approximately 10 years

  2. Number of Participants Diagnosed With NHAE nC1-INH at Each Site

    Time frame: Up to approximately 10 years

  3. Number of Participants Categorized by Type of Treatments Received by Participants

    Time frame: Up to approximately 10 years

  4. Average Dose for Each Type of Treatment Received by Participants

    Time frame: Up to approximately 10 years

  5. Average Duration of Treatment for Each Type of Treatment Received by Participants

    Time frame: Up to approximately 10 years

  6. Number of Participants Categorized by Type of Settings Where Treatment was Received

    Time frame: Up to approximately 10 years

    Categories of treatment settings include: home, hospital, physician office, emergency room visits (ER), and other.

  7. Number of Participants Receiving Multiple Long-Term Prophylaxis (LTP) Therapies at Once

    Time frame: Up to approximately 10 years

    Number of participants will be reported by number of LTP therapies received at once.

  8. Number of Participants Receiving Multiple LTP Therapies Categorized by Combination and Order of Treatments Received

    Time frame: Up to approximately 10 years

  9. Number of Attacks Occurring per Three-Months

    Time frame: Before and after initiation of treatment up to end of the study (up to approximately 10 years)

  10. Number of Participants Categorized by Severity of Attack per Three Months

    Time frame: 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.

  11. Number of Participants by Body Sites Affected by Attacks per Three Months

    Time frame: 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.

  12. Number of Participants with Symptoms of the Attack

    Time frame: Up to approximately 10 years

  13. Time from Treatment (On-demand Therapy) to Symptom Improvement/Resolution

    Time frame: Up to approximately 10 years

  14. Number of Primary Care Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH

    Time frame: Up to approximately 10 years

  15. Number of Walk-in Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH

    Time frame: Up to approximately 10 years

  16. Number of Unscheduled Physician Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH

    Time frame: Up to approximately 10 years

  17. Number of Emergency Room Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH

    Time frame: Up to approximately 10 years

  18. Number of Hospitalizations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH

    Time frame: Up to approximately 10 years

  19. Number of Intubations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH

    Time frame: Up to approximately 10 years

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

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

Acronym: PROSPECT

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
Oct 13, 2022
Registry last updated
Jul 30, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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