Hereditary Angioedema (HAE)PROSPECTNCT05578417
A Study to Review the Treatment and Outcomes of Teenagers and Adults With Non-histaminergic Angioedema With Normal C1 Inhibitor in Canada
An observational study of Hereditary Angioedema (HAE) and Angioedema, run by Takeda at 6 sites in Canada.
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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE), Angioedema
- How many people
- 60 participants
- Where
- 6 sitesCanada
- Study dates
- 15 Jun 2023to 30 Jun 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants with confirmed diagnosis of HAE nC1-INH or NHAE nC1-INH prior to January 1, 2022.
Who may be able to join
- 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:
- 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
Who cannot join
- 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
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Canada6 sites
Alberta Health Services
Calgary, Alberta, T2N 2T9
Alberta Health Services
Edmonton, Alberta, T6G 2B7
Hamilton Health Science Corporation
Hamilton, Ontario, L8S 4K1
CHU de Québec - Université Laval
Québec, G1V 4G2
Vancouver Allergy Clinic
Vancouver, British Columbia, V5Z 1H8
University of Manitoba
Winnipeg, Manitoba, R3A 1R9
About this study
Official title: 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.
Read the 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.
- Conditions
- Hereditary Angioedema (HAE), Angioedema
- Being tested
- No Intervention
- Keywords
- Drug Therapy
- Medical subject headings
- Angioedemas, Hereditary, Angioedema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
As this is an observational study, no intervention will be administered.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Diagnosed With HAE nC1-INH at Each Site
Measured: Up to approximately 10 years
Number of Participants Diagnosed With NHAE nC1-INH at Each Site
Measured: Up to approximately 10 years
Number of Participants Categorized by Type of Treatments Received by Participants
Measured: Up to approximately 10 years
Average Dose for Each Type of Treatment Received by Participants
Measured: Up to approximately 10 years
Average Duration of Treatment for Each Type of Treatment Received by Participants
Measured: 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.
Measured: 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.
Measured: Up to approximately 10 years
Number of Participants Receiving Multiple LTP Therapies Categorized by Combination and Order of Treatments Received
Measured: Up to approximately 10 years
Number of Attacks Occurring per Three-Months
Measured: 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.
Measured: 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.
Measured: 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
Measured: Up to approximately 10 years
Time from Treatment (On-demand Therapy) to Symptom Improvement/Resolution
Measured: Up to approximately 10 years
Number of Primary Care Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
Measured: Up to approximately 10 years
Number of Walk-in Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
Measured: Up to approximately 10 years
Number of Unscheduled Physician Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
Measured: Up to approximately 10 years
Number of Emergency Room Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
Measured: Up to approximately 10 years
Number of Hospitalizations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
Measured: Up to approximately 10 years
Number of Intubations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
Measured: Up to approximately 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2022
- Study start15 Jun 2023
- Primary completion31 Mar 2024
- Study completion30 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Angioedema (HAE)Studies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05578417
- Sponsor's protocol ID
- TAK-743-4013
- Acronym
- PROSPECT
- Status last verified
- Jul 2024
- First submitted
- 11 Oct 2022
- Last updated
- 30 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment and Outcomes of Patients Identified With Non-Histaminergic Angioedema With Normal C1 Inhibitor in Canada
- ClinicalTrials.gov ID
- NCT05578417
- Acronym / sponsor ID
- PROSPECT / TAK-743-4013
- Conditions
- Hereditary Angioedema (HAE), Angioedema
- Keywords
- Drug Therapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 15 Jun 2023 (actual)
- Primary completion
- 31 Mar 2024 (actual)
- Study completion
- 30 Jun 2024 (actual)
- First posted
- 13 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HAE nC1-INH | – | Other: No InterventionParticipants 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. |
| NHAE nC1-INH | – | Other: No InterventionParticipants 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. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with confirmed diagnosis of HAE nC1-INH or NHAE nC1-INH prior to January 1, 2022.
Key inclusion criteria (7 listed)
- 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:
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No InterventionAs this is an observational study, no intervention will be administered. | Other | HAE nC1-INH, NHAE nC1-INH |
Primary outcome
Number of Participants Diagnosed With HAE nC1-INH at Each Site
Time frame: Up to approximately 10 years
Number of Participants Diagnosed With NHAE nC1-INH at Each Site
Time frame: Up to approximately 10 years
Number of Participants Categorized by Type of Treatments Received by Participants
Time frame: Up to approximately 10 years
Average Dose for Each Type of Treatment Received by Participants
Time frame: Up to approximately 10 years
Average Duration of Treatment for Each Type of Treatment Received by Participants
Time frame: Up to approximately 10 years
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.
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.
Number of Participants Receiving Multiple LTP Therapies Categorized by Combination and Order of Treatments Received
Time frame: Up to approximately 10 years
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)
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.
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.
Number of Participants with Symptoms of the Attack
Time frame: Up to approximately 10 years
Time from Treatment (On-demand Therapy) to Symptom Improvement/Resolution
Time frame: Up to approximately 10 years
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
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
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
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
Number of Hospitalizations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
Time frame: Up to approximately 10 years
Number of Intubations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
Time frame: Up to approximately 10 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2024.
- To obtain more information on the study, click here/on this link.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Angioedema (HAE) and Angioedema, run by Takeda at 6 sites in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Diagnosed With HAE nC1-INH at Each Site (time frame: Up to approximately 10 years). The study lists 19 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jul 2024.