Hereditary Angioedema (HAE)NCT05469789
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)
An observational study of Hereditary Angioedema (HAE), run by Takeda at 1 site in United Kingdom.
Trial summary
The main aim of this study is to compare the HAE attacks (both total and those requiring on-demand treatment) in the 12 months before and up to 24 months after initiation of treatment with lanadelumab. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hereditary Angioedema (HAE)
- How many people
- 50 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 29 Jun 2023to 31 Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The population of this survey is all participants with HAE type I or II treated with lanadelumab in hospitals within the UK.
Who may be able to join
- Documented diagnosis of HAE type I or II (confirmed through laboratory testing)
- Documented initiation of treatment with lanadelumab or due to be initiated within next 4 weeks
- Greater than or equal to (≥)12 years of age at initiation of treatment with lanadelumab
- ≥12 months of continuous medical record data before initiation of treatment with lanadelumab
Who cannot join
- Exposure to lanadelumab administered as an investigation product in a clinical trial setting at any point in time
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Demographic and Clinical Characteristics, Treatment Patterns, and Real-world Effectiveness of Lanadelumab in Patients With Hereditary Angioedema Type I or II in the United Kingdom: an Ambispective Cohort Study
The main aim of this study is to compare the HAE attacks (both total and those requiring on-demand treatment) in the 12 months before and up to 24 months after initiation of treatment with lanadelumab.
This study is conducted in the United Kingdom where participants were treated or about to be treated with landelumab according to their routine practice at hospitals. Data will be directly collected from participants via study diaries, questionnaires, their medical records, and study doctors treating them. Participants will be contacted every 3 months during study participation (via phone).
- Conditions
- Hereditary Angioedema (HAE)
- Being tested
- No Intervention
- Keywords
- Drug therapy
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
As it is an observational study, no intervention is administered in this study.
| Study group | Role | What they receive |
|---|---|---|
| Prevalent Cohort A | Prevalent Cohort A will comprise eligible participants with HAE type I or II who have initiated treatment with lanadelumab at any point in time (i.e., all prevalent cases of exposure to lanadelumab). The total follow-up encompasses a 12-month pre-treatment period, and an up-to 24-month post-treatment period. Receives: Other: No Intervention | |
| Prevalent Cohort B | Prevalent Cohort B will comprise eligible participants with HAE type I or II who have been treated with lanadelumab for <12 months (i.e., the subset of participants from Prevalent Cohort A who initiated treatment with lanadelumab within <12 months). The total follow-up encompasses a 12-month pre-treatment and an up to 24-month post-treatment period, as well as a prospective data collection period of participant-specific duration. Receives: Other: No Intervention | |
| Incident Cohort A | Incident Cohort A will comprise eligible participants with HAE type I or II initiating treatment with lanadelumab at any time within the prospective data collection time period. The total follow-up encompasses a 12-month pre-treatment and an up-to 24-month post-treatment period, as well as a prospective data collection period of participant-specific duration. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Hereditary Angioedema (HAE) Attacks
Number of HAE attacks (both total [including untreated] and those requiring on-demand treatment) will be estimated across the pre- and post-treatment periods (that is, 12 months before and up to 24 months after initiation of treatment with lanadelumab).
Measured: Up to approximately 3 years
Secondary outcomes (16)
Number of Participants Categorized by Their Demographic Characteristics
Demographic characteristics will include age, sex, and ethnic group at index.
Measured: At index (lanadelumab treatment initiation day)
Number of Participants Categorized by Their Clinical Characteristics
Clinical characteristics will include categories of participant-reported age at onset of symptoms, age at diagnosis of HAE, duration of time from onset to diagnosis (i.e., the diagnostic delay), body mass index (BMI) (at index); comorbidities (pre and post index), history of laryngeal attacks (pre index), type of HAE (type I and II), and triggers for HAE symptoms onset (at index and post index). Index is defined as the lanadelumab treatment initiation day.
Measured: Up to approximately 3 years
Number of Participants Categorized by Their Treatment Patterns
Treatment patterns will include categories of the summary of participant-reported duration of lanadelumab treatment, treatment dose given to participants, and participant with switching patterns with lanadelumab on occurence of HAE attacks.
Measured: At and after index (lanadelumab treatment initiation day plus approximately 2 years)
Number of Participants Categorized by Their Patterns of Prophylactic Treatment
Patterns of prophylactic treatment will include categories of the summary of participant-reported use of brand and drug name, duration of treatment, dose of the treatment within same class of prophylactic treatments.
Measured: Up to approximately 3 years
Compare the Number of Participants Categorized by Their Clinical Features of HAE Attacks Before and After Initiation of Lanadelumab
To compare the number clinical features of HAE attacks before and after initiation of lanadelumab. Clinical features of HAE attacks will include categories of summary of participant-reported time of attack, duration of attack, location (e.g., trunk, extremities, and larynx) of HAE attack, the severity (Grade 1 [Mild], Grade 2 [Moderate], and Grade 3 [Severe]) of HAE attack, and hospitalization due to the HAE attack.
Measured: Up to approximately 3 years
Number of Self-Reported HAE Attacks
Measured: Up to approximately 3 years
Number of Participants Categorized by Their Clinical Features of Self-Reported HAE Attacks
Clinical features of self-reported HAE attacks will include categories of summary of participant-reported time of attack, duration of attack, location (e.g., trunk, extremities, and larynx) of HAE attack, the severity (Grade 1 [Mild], Grade 2 [Moderate], and Grade 3 [Severe]) of HAE attack, symptoms of HAE attack (e.g., tiredness, abdominal pain, and abdominal swelling), and identified or suspected trigger(s) for the attack (e.g., contact with chemicals, drugs, or foods).
Measured: Up to approximately 3 years
European Quality of Life 5 Dimensions 3-levels (EQ-5D-3L) Questionnaire Total Score
The EQ-5D-5L questionnaire will be used to record impact of HAE on participant's quality-of-life (QoL). The EQ-5D-5L is a descriptive system of Health-Related Quality-of-Life (HRQoL) states consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each of which can take 1 of 5 responses. The responses record 5 levels of severity [no problems, slight problems, moderate problems, severe problems, and extreme problems] within a particular dimension. The scores will be the sum of scores from each dimension and can range from 5 (no problems) to 25 (extreme problems). Higher score indicated a worsening health condition.
Measured: Up to approximately 2 years
EQ-5D-3L Visual Analogue Scale (VAS) Score
The EQ-5D-3L VAS records the participant's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. The VAS is used as a quantitative measure of health outcome that reflect the participant's own judgement score, with a score of 0 as the worst and 100 as the best possible health.
Measured: Up to approximately 2 years
Angioedema Quality of Life (AE-QoL) Total Scores
AE-QoL is a self-administered instrument designed to measure HRQoL in adult participants with recurrent angioedema. The AE-QoL consists of 17 disease-specific quality-of-life items, to produce a total AE-QoL score and 4 domain scores (functioning, fatigue/mood, fears/shame, and food, Each of the 17 items has a five-point response scale ranging from 1 (Never) to 5 (Very Often).
Measured: Up to approximately 2 years
Angioedema Quality of Life (AE-QoL) Domain Scores
AE-QoL is a self-administered instrument designed to measure HRQoL in adult participants with recurrent angioedema. The AE-QoL consists of 17 disease-specific quality-of-life items, to produce a total AE-QoL score and 4 domain scores (functioning, fatigue/mood, fears/shame, and food, Each of the 17 items has a five-point response scale ranging from 1 (Never) to 5 (Very Often).
Measured: Up to approximately 2 years
Number of Participants With HAE-related Health Care Resource Utilization (HCRU)
Healthcare resources used during medical encounters include in and out-patient visits to physicians, hospital admissions, in and out-patient emergency care, and in and out-patient clinical examinations and tests.
Measured: Up to approximately 3 years
Angioedema Control Test (AECT) Total Score
The AECT is used to assess participants with recurrent angioedema. The test uses a questionnaire with 4 items, each of which has 5 options and is used to measure the participant's current disease control. HAE control score will be recorded with 5 levels of answers [1=very often, 2=often, 3=sometimes, 4=seldom, 5=not at all] to the following questions: 1) In the last 3 months, how often have you had angioedema? 2) In the last 3 months, how much has your QoL been affected by angioedema? 3) In the last 3 months, how much has the unpredictability of your angioedema bothered you? 4) In the last 3 months, how well has your angioedema been controlled by your therapy?
Measured: Up to approximately 2 years
Angioedema Control Test (AECT) Domain Score
The AECT is used to assess participants with recurrent angioedema. The test uses a questionnaire with 4 items, each of which has 5 options and is used to measure the participant's current disease control. HAE control score will be recorded with 5 levels of answers [1=very often, 2=often, 3=sometimes, 4=seldom, 5=not at all] to the following questions: 1) In the last 3 months, how often have you had angioedema? 2) In the last 3 months, how much has your QoL been affected by angioedema? 3) In the last 3 months, how much has the unpredictability of your angioedema bothered you? 4) In the last 3 months, how well has your angioedema been controlled by your therapy?
Measured: Up to approximately 2 years
Treatment Satisfaction Questionnaire for Medication (TSQM) Total Score
The TSQM comprises 14 items across four domains focusing on effectiveness (three items), side effects (five items), convenience (three items), and global satisfaction (three items) of the medication over the previous 2-3 weeks, or since the patient's last use. With the exception of item 4 (presence of side effects; yes or no), all items have five or seven responses, scored from one (least satisfied) to five or seven (most satisfied). The 7-item scales had a non-neutral midpoint, such that there were more positive response options than negative response options, to allow for precise information to be obtained at the upper end of the score distribution. Item scores are summed to give four domain scores, which are in turn transformed to a scale of 0-100.
Measured: Up to approximately 2 years
Treatment Satisfaction Questionnaire for Medication (TSQM) Domain Score
The TSQM comprises 14 items across four domains focusing on effectiveness (three items), side effects (five items), convenience (three items), and global satisfaction (three items) of the medication over the previous 2-3 weeks, or since the patient's last use. With the exception of item 4 (presence of side effects; yes or no), all items have five or seven responses, scored from one (least satisfied) to five or seven (most satisfied). The 7-item scales had a non-neutral midpoint, such that there were more positive response options than negative response options, to allow for precise information to be obtained at the upper end of the score distribution. Item scores are summed to give four domain scores, which are in turn transformed to a scale of 0-100.
Measured: Up to approximately 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2022
- Study start29 Jun 2023
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
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
- Other
- Enrolment
- 50 participants (estimated)
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
- NCT05469789
- Sponsor's protocol ID
- TAK-743-0001
- Other IDs
- MACS-2021-0616012
- Status last verified
- Nov 2025
- First submitted
- 20 Jul 2022
- Last updated
- 20 Nov 2025
- 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 20 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Demographic and Clinical Characteristics, Treatment Patterns, and Real-world Effectiveness of Lanadelumab in Patients With Hereditary Angioedema Type I or II in the United Kingdom: an Ambispective Cohort Study
- ClinicalTrials.gov ID
- NCT05469789
- Acronym / sponsor ID
- TAK-743-0001
- Conditions
- Hereditary Angioedema (HAE)
- Keywords
- Drug therapy
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 29 Jun 2023 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 22 Jul 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 compare the HAE attacks (both total and those requiring on-demand treatment) in the 12 months before and up to 24 months after initiation of treatment with lanadelumab.
This study is conducted in the United Kingdom where participants were treated or about to be treated with landelumab according to their routine practice at hospitals. Data will be directly collected from participants via study diaries, questionnaires, their medical records, and study doctors treating them. Participants will be contacted every 3 months during study participation (via phone).
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Prevalent Cohort A | – | Other: No InterventionPrevalent Cohort A will comprise eligible participants with HAE type I or II who have initiated treatment with lanadelumab at any point in time (i.e., all prevalent cases of exposure to lanadelumab). The total follow-up encompasses a 12-month pre-treatment period, and an up-to 24-month post-treatment period. |
| Prevalent Cohort B | – | Other: No InterventionPrevalent Cohort B will comprise eligible participants with HAE type I or II who have been treated with lanadelumab for <12 months (i.e., the subset of participants from Prevalent Cohort A who initiated treatment with lanadelumab within <12 months). The total follow-up encompasses a 12-month pre-treatment and an up to 24-month post-treatment period, as well as a prospective data collection period of participant-specific duration. |
| Incident Cohort A | – | Other: No InterventionIncident Cohort A will comprise eligible participants with HAE type I or II initiating treatment with lanadelumab at any time within the prospective data collection time period. The total follow-up encompasses a 12-month pre-treatment and an up-to 24-month post-treatment period, as well as a prospective data collection period of participant-specific duration. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The population of this survey is all participants with HAE type I or II treated with lanadelumab in hospitals within the UK.
Key inclusion criteria (4 listed)
- Documented diagnosis of HAE type I or II (confirmed through laboratory testing)
- Documented initiation of treatment with lanadelumab or due to be initiated within next 4 weeks
- Greater than or equal to (≥)12 years of age at initiation of treatment with lanadelumab
- ≥12 months of continuous medical record data before initiation of treatment with lanadelumab
Key exclusion criteria (1 listed)
- Exposure to lanadelumab administered as an investigation product in a clinical trial setting at any point in time
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 it is an observational study, no intervention is administered in this study. | Other | Incident Cohort A, Prevalent Cohort A, Prevalent Cohort B |
Primary outcome
Number of Hereditary Angioedema (HAE) Attacks
Time frame: Up to approximately 3 years
Number of HAE attacks (both total [including untreated] and those requiring on-demand treatment) will be estimated across the pre- and post-treatment periods (that is, 12 months before and up to 24 months after initiation of treatment with lanadelumab).
16 secondary outcomes
Number of Participants Categorized by Their Demographic Characteristics
Time frame: At index (lanadelumab treatment initiation day)
Number of Participants Categorized by Their Clinical Characteristics
Time frame: Up to approximately 3 years
Number of Participants Categorized by Their Treatment Patterns
Time frame: At and after index (lanadelumab treatment initiation day plus approximately 2 years)
Number of Participants Categorized by Their Patterns of Prophylactic Treatment
Time frame: Up to approximately 3 years
Compare the Number of Participants Categorized by Their Clinical Features of HAE Attacks Before and After Initiation of Lanadelumab
Time frame: Up to approximately 3 years
Number of Self-Reported HAE Attacks
Time frame: Up to approximately 3 years
Number of Participants Categorized by Their Clinical Features of Self-Reported HAE Attacks
Time frame: Up to approximately 3 years
European Quality of Life 5 Dimensions 3-levels (EQ-5D-3L) Questionnaire Total Score
Time frame: Up to approximately 2 years
EQ-5D-3L Visual Analogue Scale (VAS) Score
Time frame: Up to approximately 2 years
Angioedema Quality of Life (AE-QoL) Total Scores
Time frame: Up to approximately 2 years
Angioedema Quality of Life (AE-QoL) Domain Scores
Time frame: Up to approximately 2 years
Number of Participants With HAE-related Health Care Resource Utilization (HCRU)
Time frame: Up to approximately 3 years
Angioedema Control Test (AECT) Total Score
Time frame: Up to approximately 2 years
Angioedema Control Test (AECT) Domain Score
Time frame: Up to approximately 2 years
Treatment Satisfaction Questionnaire for Medication (TSQM) Total Score
Time frame: Up to approximately 2 years
Treatment Satisfaction Questionnaire for Medication (TSQM) Domain Score
Time frame: Up to approximately 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2025.
- To obtain more information on the study, click here/on this linkLink 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
1 site 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), run by Takeda at 1 site in United Kingdom.
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 4 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 20 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. 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?
This study is still under way. Results are not available in the source record yet.
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 Hereditary Angioedema (HAE) Attacks (time frame: Up to approximately 3 years). 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 20 Nov 2025.