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Hereditary Angioedema (HAE)EMPOWERNCT03845400

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

An observational study of Hereditary Angioedema (HAE), run by Shire at 29 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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. Read more about this study

Who is it for?
Any age
Conditions the study is for
Hereditary Angioedema (HAE)
How many people
168 participants
Where
29 sitesin 3 countries
Study dates
30 Mar 2019to 22 Oct 2022
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages Any age
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: Participants with a diagnosis of Type I or Type II HAE.

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

29 sites in 3 countries.

29 sites in 3 countries

United States23 sites
  • University of Michigan

    Ann Arbor, Michigan, 48106

  • Riverside Medical Group, Belleville

    Belleville, New Jersey, 07109

  • Clinical Research Center of Alabama

    Birmingham, Alabama, 35209

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114-2752

  • Clinical Research of Charlotte

    Charlotte, North Carolina, 28277

  • Institute for Asthma & Allergy - Chevy Chase

    Chevy Chase, Maryland, 20815

  • Bernstein Clinical Research Center, LLC

    Cincinnati, Ohio, 45231

  • Asthma and Allergy Associates, PC

    Colorado Springs, Colorado, 80907

  • AARA Research Center

    Dallas, Texas, 75231

  • Duke Asthma, Allergy & Airway Center

    Durham, North Carolina, 27704

  • Jay M Kashkin, MD Allergy, Asthma and Immunology

    Fair Lawn, New Jersey, 07410

  • Portland Clinical Research/AAIM Care

    Happy Valley, Oregon, 97086

  • Penn State Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • University of Kansas Medical Center Research Institute, Inc.

    Kansas City, Kansas, 66160

  • Yale University School of Medicine

    New Haven, Connecticut, 06519

  • Mount Sinai

    New York, New York, 10029

  • Mid West Immunology Clinic

    Plymouth, Minnesota, 55446

  • University of California San Diego

    San Diego, California, 92122

  • AIRE Medical of Los Angeles

    Santa Monica, California, 90404

  • Medical Research of Arizona

    Scottsdale, Arizona, 85248

  • Seattle Allergy & Asthma Research Institute

    Seattle, Washington, 98115

  • Washington University

    St Louis, Missouri, 63141

  • University of South Florida Asthma, Allergy & Immunology

    Tampa, Florida, 33613

Other trials in United States

Canada5 sites
  • University of Calgary - Heritage Medical Research Clinic

    Calgary, Alberta, T2N 2T9

  • McMaster University Health Sciences Center

    Hamilton, Ontario, L8S 4K1

  • CHUM Hôtel-Dieu

    Montreal, Quebec, H2X 0A9

  • Clinique Specialisée en Allergie de la Capitale

    Québec, G1V 4W2

  • St. Michael's Hospital

    Toronto, Ontario, M5B 1W8

Other trials in Canada

Puerto Rico1 site
  • Rafael H Zaragoza-Urdaz

    San Juan, 00918

Other trials in Puerto Rico

About this study

Official title: 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.

Medical subject headings
Angioedemas, Hereditary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Study groups in NCT03845400
Study groupRoleWhat they receive
Type I or Type II HAE ParticipantsParticipants 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 being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Hereditary Angioedema (HAE) Attacks

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

    Measured: From enrollment up to 36 months

Secondary outcomes (14)
  1. Dose of Lanadelumab Injections

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

    Measured: From enrollment up to 36 months

  2. Frequency of Lanadelumab Injections

    Frequency of lanadelumab injections during the study will be reported.

    Measured: From enrollment up to 36 months

  3. 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.

    Measured: From enrollment up to 36 months

  4. Time From Diagnosis to Lanadelumab Initiation

    Time from diagnosis of HAE attack(s) to lanadelumab treatment initiation will be reported.

    Measured: From enrollment up to 36 months

  5. 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.

    Measured: From enrollment up to 36 months

  6. 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.

    Measured: From enrollment up to 36 months

  7. Number of Hospitalizations due to Hereditary Angioedema (HAE) Attack(s)

    Number of hospitalizations due to HAE attack(s) will be reported.

    Measured: From enrollment up to 36 months

  8. 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.

    Measured: From enrollment up to 36 months

  9. Number of Physician Visits due to Hereditary Angioedema (HAE) Attack(s)

    Number of physician visits due to HAE attack(s) will be reported.

    Measured: From enrollment up to 36 months

  10. 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.

    Measured: From enrollment up to 36 months

  11. 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?

    Measured: From enrollment up to 36 months (every 3 months)

  12. 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.

    Measured: From enrollment up to 36 months (every 3 months)

  13. 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.

    Measured: From enrollment up to 36 months (every 3 months)

  14. 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.

    Measured: From enrollment up to 36 months (every 3 months)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Feb 2019
  2. Study start30 Mar 2019
  3. Primary completion22 Oct 2022
  4. Study completion22 Oct 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
168 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03845400
Sponsor's protocol ID
SHP643-403
Other IDs
TAK-743-403
Acronym
EMPOWER
Status last verified
Mar 2023
First submitted
18 Feb 2019
Last updated
30 Mar 2023
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: 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.

Publications

  • Zaragoza-Urdaz RH, Busse PJ, Fox D, Castaner DN, Khutoryansky N, Juethner S, McBayne T. Lanadelumab's Enduring Effectiveness and Safety in the Management of Hereditary Angioedema for Patients from Puerto Rico: Data from EMPOWER. Int Arch Allergy Immunol. 2026 Mar 12:1-11. doi: 10.1159/000550883. Online ahead of print.

    PubMed 41818404
  • Betschel SD, Chapdelaine H, Gagnon R, Goodyear MD, Keith PK, El-Zoeiby A, Khutoryansky N, Castaner DN. Long-term effectiveness and safety of lanadelumab in Canadian patients with hereditary angioedema: a subanalysis of the EMPOWER study. Allergy Asthma Clin Immunol. 2026 Jan 21;22(1):11. doi: 10.1186/s13223-025-01007-9.

    PubMed 41566486
  • Zanichelli A, Wuillemin WA, Aygoren-Pursun E, Banerji A, Busse PJ, Betschel SD, Cancian M, Gagnon R, Goodyear MD, Kinaciyan T, Kessel A, Magerl M, Recke A, Wedner HJ, Estepan DN, Watt M, Andresen I, Juethner S, Khutoryansky N, Martinez-Saguer I. Lanadelumab's impact on hereditary angioedema control and quality of life across disease activity subgroups: Real-world evidence. Ann Allergy Asthma Immunol. 2025 Nov;135(5):560-569.e2. doi: 10.1016/j.anai.2025.07.025. Epub 2025 Sep 6.

    PubMed 40769455
  • Bernstein JA, Betschel SD, Busse PJ, Banerji A, Wedner HJ, Manning M, Zaragoza-Urdaz RH, Anderson J, Gagnon R, Baptist AP, Soteres D, Lumry WR, Craig T, Petroni D, Hsu FI, Nova Estepan D, Juethner S, Watt M, Khutoryansky N, Zuraw BL; EMPOWER Investigators. Sustained Effectiveness, Tolerability, and Safety of Long-Term Prophylaxis with Lanadelumab in Hereditary Angioedema: The Prospective, Phase 4, Noninterventional EMPOWER Real-World Study. Adv Ther. 2025 Aug;42(8):3882-3901. doi: 10.1007/s12325-025-03226-3. Epub 2025 Jun 12.

    PubMed 40504359
  • Tachdjian R, Banerji A, Busse PJ, Agmon-Levin N, Anderson J, Cancian M, Spadaro G, Enciu C, Estepan DN, Khutoryansky N, Jain S, Recke A. Effective long-term prophylaxis with lanadelumab in adolescents with hereditary angioedema: EMPOWER/ENABLE. Pediatr Allergy Immunol. 2025 Apr;36(4):e70072. doi: 10.1111/pai.70072.

    PubMed 40171989

Questions about this trial

What is this clinical trial studying?

An observational study of Hereditary Angioedema (HAE), run by Shire at 29 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 29 sites in United States, Canada, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Hereditary Angioedema (HAE) Attacks (time frame: From enrollment up to 36 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03845400, which also lists 5 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2023.

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