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

Hereditary Angioedema (HAE) Clinical Trial

— EMPOWER  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03845400
• Other study ID #: SHP643-403
• Secondary ID: TAK-743-403
• Status: Completed
• Start date: March 30, 2019
• Est. completion date: October 22, 2022

Study information

• Verified date: March 2023
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 168
• Est. completion date: October 22, 2022
• Est. primary completion date: October 22, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Locations

Country	Name	City	State
Canada	University of Calgary - Heritage Medical Research Clinic	Calgary	Alberta
Canada	McMaster University Health Sciences Center	Hamilton	Ontario
Canada	CHUM Hôtel-Dieu	Montreal	Quebec
Canada	Clinique Specialisée en Allergie de la Capitale	Québec
Canada	St. Michael's Hospital	Toronto	Ontario
Puerto Rico	Rafael H Zaragoza-Urdaz	San Juan
United States	University of Michigan	Ann Arbor	Michigan
United States	Riverside Medical Group, Belleville	Belleville	New Jersey
United States	Clinical Research Center of Alabama	Birmingham	Alabama
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Clinical Research of Charlotte	Charlotte	North Carolina
United States	Institute for Asthma & Allergy - Chevy Chase	Chevy Chase	Maryland
United States	Bernstein Clinical Research Center, LLC	Cincinnati	Ohio
United States	Asthma and Allergy Associates, PC	Colorado Springs	Colorado
United States	AARA Research Center	Dallas	Texas
United States	Duke Asthma, Allergy & Airway Center	Durham	North Carolina
United States	Jay M Kashkin, MD Allergy, Asthma and Immunology	Fair Lawn	New Jersey
United States	Portland Clinical Research/AAIM Care	Happy Valley	Oregon
United States	Penn State Hershey Medical Center	Hershey	Pennsylvania
United States	University of Kansas Medical Center Research Institute, Inc.	Kansas City	Kansas
United States	Yale University School of Medicine	New Haven	Connecticut
United States	Mount Sinai	New York	New York
United States	Mid West Immunology Clinic	Plymouth	Minnesota
United States	Washington University	Saint Louis	Missouri
United States	University of California San Diego	San Diego	California
United States	AIRE Medical of Los Angeles	Santa Monica	California
United States	Medical Research of Arizona	Scottsdale	Arizona
United States	Seattle Allergy & Asthma Research Institute	Seattle	Washington
United States	University of South Florida Asthma, Allergy & Immunology	Tampa	Florida

Sponsors (2)

Lead Sponsor	Collaborator
Shire	Takeda Development Center Americas, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Puerto Rico, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Dose of Lanadelumab Injections	Dose of lanadelumab injections used during the study will be reported.	From enrollment up to 36 months
Secondary	Frequency of Lanadelumab Injections	Frequency of lanadelumab injections during the study will be reported.	From enrollment up to 36 months
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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)
Secondary	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)
Secondary	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)
Secondary	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)

External Links

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