Clinical trial NCT03845400: A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America
Official title: An Observational, Non-interventional, Study of Patients With Hereditary Angioedema in the United States and Canada (EMPOWER Study)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hereditary Angioedema (HAE)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Takeda Development Center Americas, Inc.
First posted: 2019-02-19
Enrollment: 168
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants with a diagnosis of Type I or Type II HAE.

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Hereditary Angioedema (HAE) Attacks
timeFrame: From enrollment up to 36 months
Secondary outcomes:
measure: Dose of Lanadelumab Injections
timeFrame: From enrollment up to 36 months
measure: Frequency of Lanadelumab Injections
timeFrame: From enrollment up to 36 months
measure: Proportion of Injections Based on the Type of Administration of Lanadelumab
timeFrame: From enrollment up to 36 months
measure: Time From Diagnosis to Lanadelumab Initiation
timeFrame: From enrollment up to 36 months
measure: Proportion of Participants who Discontinue Lanadelumab
timeFrame: From enrollment up to 36 months
measure: Number of Lanadelumab Injections Before Discontinuation per Reason for Injection
timeFrame: From enrollment up to 36 months
measure: Number of Hospitalizations due to Hereditary Angioedema (HAE) Attack(s)
timeFrame: From enrollment up to 36 months
measure: Number of Emergency Room (ER) Visits due to Hereditary Angioedema (HAE) Attack(s)
timeFrame: From enrollment up to 36 months
measure: Number of Physician Visits due to Hereditary Angioedema (HAE) Attack(s)
timeFrame: From enrollment up to 36 months
measure: Rescue Medications Taken at Time of Hereditary Angioedema (HAE) Attack(s)
timeFrame: From enrollment up to 36 months
measure: Hereditary Angioedema (HAE) Attack Control Score Before and After Lanadelumab Initiation, as Measured by the Angioedema Control Test (AECT)
timeFrame: From enrollment up to 36 months (every 3 months)
measure: Angioedema Quality of Life (AE-QoL) Score
timeFrame: From enrollment up to 36 months (every 3 months)
measure: Work Productivity and Activity Impairment: General Health (WPAI:GH) Score
timeFrame: From enrollment up to 36 months (every 3 months)
measure: Treatment Satisfaction (TSQM-9) Score
timeFrame: From enrollment up to 36 months (every 3 months)
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Clinical Research Center of Alabama
state: Alabama
city: Scottsdale
country: United States
facility: Medical Research of Arizona
state: Arizona
city: San Diego
country: United States
facility: University of California San Diego
state: California
city: Santa Monica
country: United States
facility: AIRE Medical of Los Angeles
state: California
city: Colorado Springs
country: United States
facility: Asthma and Allergy Associates, PC
state: Colorado
city: New Haven
country: United States
facility: Yale University School of Medicine
state: Connecticut
city: Tampa
country: United States
facility: University of South Florida Asthma, Allergy & Immunology
state: Florida
city: Kansas City
country: United States
facility: University of Kansas Medical Center Research Institute, Inc.
state: Kansas
city: Chevy Chase
country: United States
facility: Institute for Asthma & Allergy - Chevy Chase
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Plymouth
country: United States
facility: Mid West Immunology Clinic
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Belleville
country: United States
facility: Riverside Medical Group, Belleville
state: New Jersey
city: Fair Lawn
country: United States
facility: Jay M Kashkin, MD Allergy, Asthma and Immunology
state: New Jersey
city: New York
country: United States
facility: Mount Sinai
state: New York
city: Charlotte
country: United States
facility: Clinical Research of Charlotte
state: North Carolina
city: Durham
country: United States
facility: Duke Asthma, Allergy & Airway Center
state: North Carolina
city: Cincinnati
country: United States
facility: Bernstein Clinical Research Center, LLC
state: Ohio
city: Happy Valley
country: United States
facility: Portland Clinical Research/AAIM Care
state: Oregon
city: Hershey
country: United States
facility: Penn State Hershey Medical Center
state: Pennsylvania
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: Seattle
country: United States
facility: Seattle Allergy & Asthma Research Institute
state: Washington
city: Calgary
country: Canada
facility: University of Calgary - Heritage Medical Research Clinic
state: Alberta
city: Hamilton
country: Canada
facility: McMaster University Health Sciences Center
state: Ontario
city: Toronto
country: Canada
facility: St. Michael's Hospital
state: Ontario
city: Montreal
country: Canada
facility: CHUM Hôtel-Dieu
state: Quebec
city: Québec
country: Canada
facility: Clinique Specialisée en Allergie de la Capitale
city: San Juan
country: Puerto Rico
facility: Rafael H Zaragoza-Urdaz
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: