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

NCT04130191 | Completed

• 2 other identifiers: SHP643-402TAK-743-402
• Study Type: observational
• Target: 140
• Locations: 7 countries
• Sites: 19

Brief Summary

The main aim of this study is to compare the number of HAE attacks occuring in persons using lanadelumab with the number of HAE attacks before lanadelumab treatment was started. Data from participants who start the study after 1 March 2021, will be collected for 24 months; data from all other participants (who started the study before 1 March 2021) will be collected for 36 months. Participants will report information in a smartphone application at study start and for the next 3 months and then every 6 months until the study ends; data will also be collected by the study doctor during routine clinic visits

Conditions

Hereditary Angioedema (HAE)

Outcome Measures

Primary Outcomes (1)

• Incidence Rate Ratio of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks

  HAE attack is defined as a discrete episode during which the participant progress from no angioedema to symptoms of angioedema. Incidence rate ratio of on-treatment patient-reported HAE attacks up to three years after initiation of lanadelumab compared to the history of HAE attacks of last three months prior to lanadelumab use will be reported.

  Up to 36 months

Secondary Outcomes (21)

• Rate of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks From Day 70

  From Day 70 up to 36 months

• Rate of Mild, Moderate, Severe Hereditary Angioedema (HAE) Attacks

  Up to 36 months

• Number of On-Treatment Participant-Reported Hereditary Angioedema (HAE) Attacks Based on Anatomical Location

  Up to 36 months

• Proportion of Hereditary Angioedema (HAE) Attacks for Which On-Demand Therapy is Used

  Up to 36 months

• Time to First Hereditary Angioedema (HAE) Attack for Which On-Demand Therapy is Used

  Up to 36 months

Study Arms (1)

Participants with hereditary angioedema (HAE)

Participants who initiate treatment with lanadelumab according to current product labelling will be enrolled and followed for up to 24 or 36 months (depending on their enrollment date).

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Participants with type 1 or type 2 HAE who initiate treatment with lanadelumab according to the current product labelling.

You may qualify if:

• Voluntarily provision of written, signed, and dated (personally or via a legally authorized representative) informed consent to participate in the study.
• Initiation of treatment with lanadelumab in accordance with current product labelling. Decision to start treatment with lanadelumab must be made before and be independent from enrollment in the study.
• Availability of information for HAE attack-related variables assessed at study enrollment for the previous three months.
• Ability to use a smartphone for data collection in this study.

You may not qualify if:

• Inability to provide written, signed, and dated informed consent.
• Participation in the study not considered appropriate by the treating physician/investigator.
• Contraindication to treatment with lanadelumab according to current product labelling.
• Pregnant or breastfeeding.
• Concomitant exposure to any investigational therapy (including medications not used for HAE).
• Current or planned participation in other interventional studies.

Sponsors & Collaborators

• Shire: lead
• Takeda Development Center Americas, Inc.: collaborator

Study Sites (19)

Medical University of Vienna

Vienna, 1090, Austria

Location

Klinikum der Johann-Wolfgang Goethe-Universitat

Frankfurt am Main, Hesse, 60590, Germany

Location

Charité - Universitätsmedizin Berlin

Berlin, 10117, Germany

Location

Universitatsklinikum Dusseldorf

Düsseldorf, 40225, Germany

Location

Universitatsklinikum Schleswig-Holstein

Lübeck, 23538, Germany

Location

Hämophilie Zentrum Rhein Main GmbH

Mörfelden-Walldorf, 64546, Germany

Location

Klinikum rechts der Isar der Technischen Universität München

München, 81675, Germany

Location

Universitätsklinikum Münster

Münster, 48149, Germany

Location

Universitätsklinikum Ulm

Ulm, 89075, Germany

Location

Bnai Zion Medical Center

Haifa, 31048, Israel

Location

Sheba Medical Center - PPDS

Ramat Gan, 52621, Israel

Location

Azienda Ospedaliera Universitaria Federico II

Napoli, Campania, 80131, Italy

Location

ASST Fatebenefratelli Sacco - Ospedale Luigi Sacco

Milan, 20157, Italy

Location

Azienda Ospedale Università Padova

Padua, 35128, Italy

Location

Al-Rashed Allergy Center

Kuwait City, 90806, Kuwait

Location

Hospital Universitario de Bellvitge

L'Hospitalet de Llobregat, Barcelona, 08907, Spain

Location

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, 08035, Spain

Location

Universitätsspital Basel

Basel, 4031, Switzerland

Location

Luzerner Kantonsspital LUKS

Lucerne, 6000, Switzerland

Location

Related Publications (2)

• 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.

  PMID: 40769455 DERIVED

• 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.

  PMID: 40171989 DERIVED

Related Links

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

MeSH Terms

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: October 3, 2019
• First Posted: October 17, 2019
• Study Start: December 11, 2019
• Primary Completion: September 9, 2024
• Study Completion: September 9, 2024
• Last Updated: September 2, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share
• 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.

Locations

AU (1); IT (3); ES (2); CH (2); KU (1); IL (2); DE (8)