A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)
Study Details
Study Description
Brief Summary
The main aim of this study is to compare the HAE attack rate requiring on-demand treatment before and within 2 years after participants with HAE have been treated 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).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| 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. |
Other: No Intervention
As it is an observational study, no intervention is administered in this study.
|
| 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. |
Other: No Intervention
As it is an observational study, no intervention is administered in this study.
|
| 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. |
Other: No Intervention
As it is an observational study, no intervention is administered in this study.
|
Outcome Measures
Primary Outcome Measures
- Number of Hereditary Angioedema (HAE) Attacks Requiring On-demand Treatment [Up to approximately 3 years]
Number of HAE attacks requiring on-demand treatment before and after the index date (lanadelumab treatment initiation day) will be reported.
Secondary Outcome Measures
- Number of Participants Categorized by Their Demographic Characteristics [At index (lanadelumab treatment initiation day)]
Demographic characteristics will include age, sex, and ethnic group at index.
- Number of Participants Categorized by Their Clinical Characteristics [Up to approximately 3 years]
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.
- Number of Participants Categorized by Their Treatment Patterns [At and after index (lanadelumab treatment initiation day plus approximately 2 years)]
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.
- Number of Participants Categorized by Their Patterns of Prophylactic Treatment [Up to approximately 3 years]
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.
- Number of Participants Categorized by Their Clinical Features of HAE Attacks Requiring On-Demand Treatment [Up to approximately 3 years]
Clinical features of HAE attacks requiring on-demand treatment 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.
- Number of Self-Reported HAE Attacks [Up to approximately 3 years]
- Number of Participants Categorized by Their Clinical Features of Self-Reported HAE Attacks [Up to approximately 3 years]
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).
- European Quality of Life 5 Dimensions 3-levels (EQ-5D-3L) Questionnaire Total Score [Up to approximately 2 years]
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.
- EQ-5D-3L Visual Analogue Scale (VAS) Score [Up to approximately 2 years]
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.
- Hereditary Angioedema Quality of Life (HAE-QoL) Total Scores [Up to approximately 2 years]
HAE-QoL is a self-administered instrument designed to measure HRQoL in adult participants with HAE type I and II.The AE-QoL consists of 25 disease-specific quality-of-life items, to produce a total AE-QoL score and 7 domain scores (treatment difficulties, physical functioning and health, disease-related stigma, emotional role and social functioning, concern about offspring, perceived control over illness, and mental health) and each of the 25 items has a five-point response scale ranging from 1 (Never) to 5 (Very Often).
- Hereditary Angioedema Quality of Life (HAE-QoL) Domain Scores [Up to approximately 2 years]
HAE-QoL is a self-administered instrument designed to measure HRQoL in adult participants with HAE type I and II.The AE-QoL consists of 25 disease-specific quality-of-life items, to produce a total AE-QoL score and 7 domain scores (treatment difficulties, physical functioning and health, disease-related stigma, emotional role and social functioning, concern about offspring, perceived control over illness, and mental health) and each of the 25 items has a five-point response scale ranging from 1 (Never) to 5 (Very Often).
- Number of Participants With HAE-related Health Care Resource Utilization (HCRU) [Up to approximately 3 years]
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.
- Angioedema Control Test (AECT) Total Score [Up to approximately 2 years]
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?
- Angioedema Control Test (AECT) Domain Score [Up to approximately 2 years]
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?
- Treatment Satisfaction Questionnaire for Medication (TSQM) Total Score [Up to approximately 2 years]
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.
- Treatment Satisfaction Questionnaire for Medication (TSQM) Domain Score [Up to approximately 2 years]
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.
Eligibility Criteria
Criteria
Inclusion:
-
Documented diagnosis of HAE type I or II (confirmed through laboratory testing)
-
Documented initiation of treatment with lanadelumab
-
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
Exclusion:
• Exposure to lanadelumab administered as an investigation product in a clinical trial setting at any point in time
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Barts Health NHS Trust | London | England | United Kingdom | E1 1BB |
Sponsors and Collaborators
- Takeda
Investigators
- Study Director: Study Director, Takeda
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- TAK-743-0001
- MACS-2021-0616012