A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America

Study Overview
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Brief Summary
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The main aim of this study is to compare the HAE attack rate before and after lanadelumab treatment was started in persons with Hereditary Angioedeme Type I or II. Data from participants will be collected for at least 24 months. Participants will report information in a smartphone application at study start and then every 3 months until the study ends; data will also be collected by the study doctor during routine clinic visits.

Official Title
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An Observational, Non-interventional, Study of Patients With Hereditary Angioedema in the United States and Canada (EMPOWER Study)

Conditions
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Hereditary Angioedema (HAE)

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Voluntarily provide written, signed, and dated (personally or via a legally-authorized representative) informed consent/and assent as applicable to participate in the study. Expression of understanding and agreement by fully informed parent(s) or legal guardian is required to permit the investigator to enroll a child in this study. The choice of the terms parental consent or parental permission in different regions may reflect local legal/regulatory and ethical considerations. Diagnosis of HAE Type I or Type II. Ability to use a mobile device for data collection in the study. Exclusion Criteria: Participation in any interventional clinical trial at the time of enrollment. Unable to provide written, signed, and dated informed consent/assent. Investigator believes that the participant is not a suitable candidate for the study.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Type I or Type II HAE Participants<br>Participants with Type I or Type II HAE will be followed for 24 or 36 months depending upon enrollment date. Data collection will cease at the end of follow-up period, at the time of withdrawal, lost to follow-up, or death whichever comes first. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Hereditary Angioedema (HAE) Attacks | Number of HAE attacks before and after lanadelumab initiation will be reported. | From enrollment up to 36 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Dose of Lanadelumab Injections | Dose of lanadelumab injections used during the study will be reported. | From enrollment up to 36 months | 
| Frequency of Lanadelumab Injections | Frequency of lanadelumab injections during the study will be reported. | From enrollment up to 36 months | 
| Proportion of Injections Based on the Type of Administration of Lanadelumab | Proportion of injections based on the type of administration of lanadelumab (self, caregiver, health care provider [HCP], other) will be reported. | From enrollment up to 36 months | 
| Time From Diagnosis to Lanadelumab Initiation | Time from diagnosis of HAE attack(s) to lanadelumab treatment initiation will be reported. | From enrollment up to 36 months | 
| Proportion of Participants who Discontinue Lanadelumab | Discontinuation is defined as no documented lanadelumab use after at least 2 missing consecutive doses after the last date of injection, or documented as treatment discontinued by the physician. Proportion of participants who discontinue lanadelumab treatment will be reported. | From enrollment up to 36 months | 
| Number of Lanadelumab Injections Before Discontinuation per Reason for Injection | Discontinuation is defined as no documented lanadelumab use after at least 2 missing consecutive doses after the last date of injection, or documented as treatment discontinued by the physician. Number of injections administered by participant before discontinuation per reason for injection will be reported. | From enrollment up to 36 months | 
| Number of Hospitalizations due to Hereditary Angioedema (HAE) Attack(s) | Number of hospitalizations due to HAE attack(s) will be reported. | From enrollment up to 36 months | 
| Number of Emergency Room (ER) Visits due to Hereditary Angioedema (HAE) Attack(s) | Number of emergency room (ER) visits due to HAE attack(s) will be reported. | From enrollment up to 36 months | 
| Number of Physician Visits due to Hereditary Angioedema (HAE) Attack(s) | Number of physician visits due to HAE attack(s) will be reported. | From enrollment up to 36 months | 
| Rescue Medications Taken at Time of Hereditary Angioedema (HAE) Attack(s) | Number, type of rescue medications taken by the participants at the time of HAE attack(s) per the type of administration (self, caregiver, HCP, other) will be reported. | From enrollment up to 36 months | 
| Hereditary Angioedema (HAE) Attack Control Score Before and After Lanadelumab Initiation, as Measured by the Angioedema Control Test (AECT) | AECT is a questionnaire and not a validated patient-reported outcome (PRO). HAE control score is evaluated as frequency of answers [very often, often, sometimes, seldom, not at all] to the following questions at record closest to enrollment date): 1. In the last 3 months, how often have you had angioedema? 2. In the last 3 months, how much has your quality of life 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? | From enrollment up to 36 months (every 3 months) | 
| Angioedema Quality of Life (AE-QoL) Score | The AE-QoL is developed to measure health-related quality of life (HRQoL) impairment in participants with recurrent angioedema. It is a self-administered PRO designed for adults aged 18 years and older with a recall period of 4 weeks. There are 17 items across 4 domains: functioning (4 items), fatigue/mood (5 items), fears/shame (6 items), and food (2 items). Responses use a 5-point Likert scale ranging from 'never' to 'very often.' Global scores range from 0 to 100 and scores by domains range from 0 to 100. | From enrollment up to 36 months (every 3 months) | 
| Work Productivity and Activity Impairment: General Health (WPAI:GH) Score | The WPAI:GH is a generic questionnaire to measure the effect of general health and symptom severity on work productivity and regular activities during the past 7 days. It can be self- or interviewer-administered to adults aged 18 years or older. This 6-item PRO covers work (5 items) and daily activities (1 item) using yes/no or numerical answers (number of hours). WPAI:GH outcomes are expressed as impairment percentages. An overall work productivity score (health or symptom) [%0WP], is calculated by multiplying the percentage of work time spent working (health or symptom) [% WTW] by the percentage productivity at work (health or symptom) [%PW]: %0WP = %WTW * %PW. | From enrollment up to 36 months (every 3 months) | 
| Treatment Satisfaction (TSQM-9) Score | The TSQM is a generic questionnaire to measure participants' satisfaction with medication using yes/no and 5- or 7- point Likert scale response options. It is a self-administered PRO designed for adults aged 18 years or older with a recall period of 2 to 3 weeks, or since the last medication use. Version TSQM-9 includes 3 domains: effectiveness (3 items), convenience (3 items), and global satisfaction scale (3 items). Scores range from 0 to 100. | From enrollment up to 36 months (every 3 months) |