A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II in North America
- Conditions
- Hereditary Angioedema (HAE)
- Registration Number
- NCT03845400
- Lead Sponsor
- Shire
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 168
- 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.
- 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 & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Hereditary Angioedema (HAE) Attacks From enrollment up to 36 months Number of HAE attacks before and after lanadelumab initiation will be reported.
- Secondary Outcome Measures
Name Time Method Time From Diagnosis to Lanadelumab Initiation From enrollment up to 36 months Time from diagnosis of HAE attack(s) to lanadelumab treatment initiation will be reported.
Number of Hospitalizations due to Hereditary Angioedema (HAE) Attack(s) From enrollment up to 36 months Number of hospitalizations due to HAE attack(s) will be reported.
Rescue Medications Taken at Time of Hereditary Angioedema (HAE) Attack(s) From enrollment up to 36 months 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.
Dose of Lanadelumab Injections From enrollment up to 36 months Dose of lanadelumab injections used during the study will be reported.
Frequency of Lanadelumab Injections From enrollment up to 36 months Frequency of lanadelumab injections during the study will be reported.
Number of Lanadelumab Injections Before Discontinuation per Reason for Injection From enrollment up to 36 months 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.
Number of Emergency Room (ER) Visits due to Hereditary Angioedema (HAE) Attack(s) From enrollment up to 36 months Number of emergency room (ER) visits due to HAE attack(s) will be reported.
Angioedema Quality of Life (AE-QoL) Score From enrollment up to 36 months (every 3 months) 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.
Work Productivity and Activity Impairment: General Health (WPAI:GH) Score From enrollment up to 36 months (every 3 months) 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.
Proportion of Participants who Discontinue Lanadelumab From enrollment up to 36 months 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.
Treatment Satisfaction (TSQM-9) Score From enrollment up to 36 months (every 3 months) 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.
Proportion of Injections Based on the Type of Administration of Lanadelumab From enrollment up to 36 months Proportion of injections based on the type of administration of lanadelumab (self, caregiver, health care provider \[HCP\], other) will be reported.
Number of Physician Visits due to Hereditary Angioedema (HAE) Attack(s) From enrollment up to 36 months Number of physician visits due to HAE attack(s) will be reported.
Hereditary Angioedema (HAE) Attack Control Score Before and After Lanadelumab Initiation, as Measured by the Angioedema Control Test (AECT) From enrollment up to 36 months (every 3 months) 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?
Trial Locations
- Locations (29)
Mount Sinai
🇺🇸New York, New York, United States
McMaster University Health Sciences Center
🇨🇦Hamilton, Ontario, Canada
Portland Clinical Research/AAIM Care
🇺🇸Happy Valley, Oregon, United States
AARA Research Center
🇺🇸Dallas, Texas, United States
Clinical Research of Charlotte
🇺🇸Charlotte, North Carolina, United States
Penn State Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
St. Michael's Hospital
🇨🇦Toronto, Ontario, Canada
Duke Asthma, Allergy & Airway Center
🇺🇸Durham, North Carolina, United States
University of Calgary - Heritage Medical Research Clinic
🇨🇦Calgary, Alberta, Canada
Rafael H Zaragoza-Urdaz
🇵🇷San Juan, Puerto Rico
Bernstein Clinical Research Center, LLC
🇺🇸Cincinnati, Ohio, United States
Seattle Allergy & Asthma Research Institute
🇺🇸Seattle, Washington, United States
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
Yale University School of Medicine
🇺🇸New Haven, Connecticut, United States
Asthma and Allergy Associates, PC
🇺🇸Colorado Springs, Colorado, United States
Mid West Immunology Clinic
🇺🇸Plymouth, Minnesota, United States
Institute for Asthma & Allergy - Chevy Chase
🇺🇸Chevy Chase, Maryland, United States
Washington University
🇺🇸Saint Louis, Missouri, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
University of Kansas Medical Center Research Institute, Inc.
🇺🇸Kansas City, Kansas, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Riverside Medical Group, Belleville
🇺🇸Belleville, New Jersey, United States
Jay M Kashkin, MD Allergy, Asthma and Immunology
🇺🇸Fair Lawn, New Jersey, United States
CHUM Hôtel-Dieu
🇨🇦Montreal, Quebec, Canada
Clinique Specialisée en Allergie de la Capitale
🇨🇦Québec, Canada
University of South Florida Asthma, Allergy & Immunology
🇺🇸Tampa, Florida, United States