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

Shire168 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

168

Conditions

Hereditary Angioedema (HAE)

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (29)

Outcomes

Primary Outcomes

Number of Hereditary Angioedema (HAE) Attacks

Number of HAE attacks before and after lanadelumab initiation will be reported.

Time frame: From enrollment up to 36 months

Secondary Outcomes

Dose of Lanadelumab Injections

Dose of lanadelumab injections used during the study will be reported.

Time frame: From enrollment up to 36 months

Frequency of Lanadelumab Injections

Frequency of lanadelumab injections during the study will be reported.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: From enrollment up to 36 months

Number of Lanadelumab Injections Before Discontinuation per Reason for Injection

Data from ClinicalTrials.gov

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Clinical Research Center of Alabama

Birmingham, Alabama, United States

Medical Research of Arizona

Scottsdale, Arizona, United States

University of California San Diego

San Diego, California, United States

AIRE Medical of Los Angeles

Santa Monica, California, United States

Asthma and Allergy Associates, PC

Colorado Springs, Colorado, United States

Yale University School of Medicine

New Haven, Connecticut, United States

University of South Florida Asthma, Allergy & Immunology

Tampa, Florida, United States

University of Kansas Medical Center Research Institute, Inc.

Kansas City, Kansas, United States

Institute for Asthma & Allergy - Chevy Chase

Chevy Chase, Maryland, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

...and 19 more locations

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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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?

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: From enrollment up to 36 months (every 3 months)