NCT03845400

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
168

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Mar 2019

Typical duration for all trials

Geographic Reach
3 countries

29 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 18, 2019

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 19, 2019

Completed
1 month until next milestone

Study Start

First participant enrolled

March 30, 2019

Completed
3.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 22, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 22, 2022

Completed
Last Updated

March 30, 2023

Status Verified

March 1, 2023

Enrollment Period

3.6 years

First QC Date

February 18, 2019

Last Update Submit

March 29, 2023

Conditions

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (14)

  • Dose of Lanadelumab Injections

    From enrollment up to 36 months

  • Frequency of Lanadelumab Injections

    From enrollment up to 36 months

  • Proportion of Injections Based on the Type of Administration of Lanadelumab

    From enrollment up to 36 months

  • Time From Diagnosis to Lanadelumab Initiation

    From enrollment up to 36 months

  • Proportion of Participants who Discontinue Lanadelumab

    From enrollment up to 36 months

  • +9 more secondary outcomes

Study Arms (1)

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.

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

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

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (29)

Clinical Research Center of Alabama

Birmingham, Alabama, 35209, United States

Location

Medical Research of Arizona

Scottsdale, Arizona, 85248, United States

Location

University of California San Diego

San Diego, California, 92122, United States

Location

AIRE Medical of Los Angeles

Santa Monica, California, 90404, United States

Location

Asthma and Allergy Associates, PC

Colorado Springs, Colorado, 80907, United States

Location

Yale University School of Medicine

New Haven, Connecticut, 06519, United States

Location

University of South Florida Asthma, Allergy & Immunology

Tampa, Florida, 33613, United States

Location

University of Kansas Medical Center Research Institute, Inc.

Kansas City, Kansas, 66160, United States

Location

Institute for Asthma & Allergy - Chevy Chase

Chevy Chase, Maryland, 20815, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114-2752, United States

Location

University of Michigan

Ann Arbor, Michigan, 48106, United States

Location

Mid West Immunology Clinic

Plymouth, Minnesota, 55446, United States

Location

Washington University

St Louis, Missouri, 63141, United States

Location

Riverside Medical Group, Belleville

Belleville, New Jersey, 07109, United States

Location

Jay M Kashkin, MD Allergy, Asthma and Immunology

Fair Lawn, New Jersey, 07410, United States

Location

Mount Sinai

New York, New York, 10029, United States

Location

Clinical Research of Charlotte

Charlotte, North Carolina, 28277, United States

Location

Duke Asthma, Allergy & Airway Center

Durham, North Carolina, 27704, United States

Location

Bernstein Clinical Research Center, LLC

Cincinnati, Ohio, 45231, United States

Location

Portland Clinical Research/AAIM Care

Happy Valley, Oregon, 97086, United States

Location

Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

AARA Research Center

Dallas, Texas, 75231, United States

Location

Seattle Allergy & Asthma Research Institute

Seattle, Washington, 98115, United States

Location

University of Calgary - Heritage Medical Research Clinic

Calgary, Alberta, T2N 2T9, Canada

Location

McMaster University Health Sciences Center

Hamilton, Ontario, L8S 4K1, Canada

Location

St. Michael's Hospital

Toronto, Ontario, M5B 1W8, Canada

Location

CHUM Hôtel-Dieu

Montreal, Quebec, H2X 0A9, Canada

Location

Clinique Specialisée en Allergie de la Capitale

Québec, G1V 4W2, Canada

Location

Rafael H Zaragoza-Urdaz

San Juan, 00918, Puerto Rico

Location

Related Publications (4)

  • 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. doi: 10.1186/s13223-025-01007-9. Online ahead of print.

  • 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.

  • 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.

  • 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.

Related Links

MeSH Terms

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

AngioedemaVascular DiseasesCardiovascular DiseasesHereditary Complement Deficiency DiseasesPrimary Immunodeficiency DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesUrticariaSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesHypersensitivity, ImmediateHypersensitivityImmune System DiseasesImmunologic Deficiency Syndromes

Study Officials

  • Study Director

    Shire

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 18, 2019

First Posted

February 19, 2019

Study Start

March 30, 2019

Primary Completion

October 22, 2022

Study Completion

October 22, 2022

Last Updated

March 30, 2023

Record last verified: 2023-03

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
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.
More information

Locations