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

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

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 Overview

• Status: Completed
• Conditions: Hereditary Angioedema (HAE)

• Study Type: Observational
• Enrollment (Actual): 168

Contacts and Locations

Study Locations

• Canada
  • Québec, Canada, G1V 4W2
    • Clinique Specialisee en Allergie de la Capitale
  • Alberta
    • Calgary, Alberta, Canada, T2N 2T9
      • University of Calgary - Heritage Medical Research Clinic
  • Ontario
    • Hamilton, Ontario, Canada, L8S 4K1
      • McMaster University Health Sciences Center
    • Toronto, Ontario, Canada, M5B 1W8
      • St. Michael's Hospital
  • Quebec
    • Montreal, Quebec, Canada, H2X 0A9
      • CHUM Hôtel-Dieu
• Puerto Rico
  • San Juan, Puerto Rico, 00918
    • Rafael H Zaragoza-Urdaz
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35209
      • Clinical Research Center of Alabama
  • Arizona
    • Scottsdale, Arizona, United States, 85248
      • Medical Research of Arizona
  • California
    • San Diego, California, United States, 92122
      • University of California San Diego
    • Santa Monica, California, United States, 90404
      • AIRE Medical of Los Angeles
  • Colorado
    • Colorado Springs, Colorado, United States, 80907
      • Asthma and Allergy Associates, PC
  • Connecticut
    • New Haven, Connecticut, United States, 06519
      • Yale University School of Medicine
  • Florida
    • Tampa, Florida, United States, 33613
      • University of South Florida Asthma, Allergy & Immunology
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center Research Institute, Inc.
  • Maryland
    • Chevy Chase, Maryland, United States, 20815
      • Institute for Asthma & Allergy - Chevy Chase
  • Massachusetts
    • Boston, Massachusetts, United States, 02114-2752
      • Massachusetts General Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48106
      • University of Michigan
  • Minnesota
    • Plymouth, Minnesota, United States, 55446
      • Mid West Immunology Clinic
  • Missouri
    • Saint Louis, Missouri, United States, 63141
      • Washington University
  • New Jersey
    • Belleville, New Jersey, United States, 07109
      • Riverside Medical Group, Belleville
    • Fair Lawn, New Jersey, United States, 07410
      • Jay M Kashkin, MD Allergy, Asthma and Immunology
  • New York
    • New York, New York, United States, 10029
      • Mount Sinai
  • North Carolina
    • Charlotte, North Carolina, United States, 28277
      • Clinical Research of Charlotte
    • Durham, North Carolina, United States, 27704
      • Duke Asthma, Allergy & Airway Center
  • Ohio
    • Cincinnati, Ohio, United States, 45231
      • Bernstein Clinical Research Center, LLC
  • Oregon
    • Happy Valley, Oregon, United States, 97086
      • Portland Clinical Research/AAIM Care
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Hershey Medical Center
  • Texas
    • Dallas, Texas, United States, 75231
      • AARA Research Center
  • Washington
    • Seattle, Washington, United States, 98115
      • Seattle Allergy & Asthma Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Participants with a diagnosis of Type I or Type II HAE.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Type I or Type II HAE Participants; 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?

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)

Collaborators and Investigators

• Sponsor: Shire
• Collaborators: Takeda Development Center Americas, Inc.

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): March 30, 2019
• Primary Completion (Actual): October 22, 2022
• Study Completion (Actual): October 22, 2022

Study Registration Dates

• First Submitted: February 18, 2019
• First Submitted That Met QC Criteria: February 18, 2019
• First Posted (Actual): February 19, 2019

Study Record Updates

• Last Update Posted (Actual): March 30, 2023
• Last Update Submitted That Met QC Criteria: March 29, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Skin Diseases; Immunologic Deficiency Syndromes; Immune System Diseases; Hypersensitivity, Immediate; Genetic Diseases, Inborn; Skin Diseases, Vascular; Hypersensitivity; Urticaria; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Angioedema; Angioedemas, Hereditary
• Other Study ID Numbers: SHP643-403; TAK-743-403 (Other Identifier: Takeda Development Center Americas)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No