A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries
A Multi-national Real-world Outcomes and Treatment Patterns Study of Lanadelumab (Takhzyro) in Paediatric Patients With Hereditary Angioedema (TAHORA)
1 other identifier
observational
40
6 countries
24
Brief Summary
HAE is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency). This study will concentrate on children with HAE C1-INH deficiency who have received Takhzyro (Lanadelumab) as prophylactic treatment. The main goal of the study is to assess how well lanadelumab works in children with HAE-C1INH deficiency in everyday life. This will be measured by checking how long children who receive lanadelumab will be free of HAE attacks. Other goals are to understand how children with HAE-C1INH deficiency are being treated with lanadelumab, how well the treatment works for them, how safe it is and how often these children need to use healthcare services (like doctor visits, hospital stays, etc.) because of their condition. The study will only look at data already existing in the participants' medical records. No treatment will be given as part of the study.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Feb 2026
Shorter than P25 for all trials
24 active sites
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
November 19, 2025
CompletedFirst Posted
Study publicly available on registry
November 26, 2025
CompletedStudy Start
First participant enrolled
February 5, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 15, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 15, 2026
May 5, 2026
April 1, 2026
4 months
November 19, 2025
April 29, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage of Participants who are Free of HAE Attacks
Percentage of participants who are free of HAE attacks will be reported. A HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx).
Up to 13 months
Secondary Outcomes (13)
HAE Attack Rate of Participants
Up to 13 months
HAE Attack-free Rate of Participants Relative to Prior Treatment
Up to 13 months
Association Between Covariates of Interest and Attack Free Rate
Up to 13 months
Number of Participants With HAE Attack Based on Attack Severity
Up to 13 months
Number of Participants With HAE Attack Based on Location
Up to 13 months
- +8 more secondary outcomes
Study Arms (1)
Participants with HAE-C1INH
Paediatric participants with HAE-C1INH who initiated long-term prophylaxis (LTP) treatment with lanadelumab within a routine clinical setting will be included.
Interventions
Eligibility Criteria
Paediatric participants who have been diagnosed with HAE-C1INH in Germany, France, United Kingdom (UK), Israel, Argentina and Serbia.
You may qualify if:
- Participant is aged 2 to less than (\<) 12 years at the time of lanadelumab initiation and is expected to have at least 6 months of follow-up information before turning 12.
- Participant has physician-confirmed diagnosis of HAE-C1INH.
- Participant initiated LTP with lanadelumab during the eligibility period.
- Signed consent/assent (where required by local regulations).
- Participant's medical record contains documentation of HAE attacks in the pre-index period and after lanadelumab initiation.
You may not qualify if:
- Participant was enrolled in a therapeutic investigational drug (lanadelumab or other drug) or device trial at index date.
- Participant with no documented HAE attacks in the 12 months prior to index date.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Takedalead
Study Sites (24)
Hospital de Ninos Sor Maria Ludovica
La Plata, Buenos Aires, B1904CSI, Argentina
Centro de Alergia e Inmunologia Clinica Mar del Plata
Mar del Plata, Buenos Aires, 7600, Argentina
Hospital de Pediatria S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Ciudad de Buenos Aires, Buenos Aires F.D., C1249ABN, Argentina
Hospital Italiano de Buenos Aires
Buenos Aires, Ciudad Autonoma de BuenosAires, C1199ABB, Argentina
Sanatorio de la Canada-Cordoba
Córdoba, X5000BSQ, Argentina
AP-HM- Hopital de La Timone
Marseille, Bouches-du-Rhone, 13005, France
CHU de Grenoble Alpes - Hopital Couple-Enfant
La Tronche, Grenoble, 38700, France
AP-HP - Hopital Armand Trousseau
Paris, 75012, France
Universitatsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081, Germany
Universitatsklinikum Frankfurt am Main
Frankfurt am Main, Hesse, 60590, Germany
Hamophilie Zentrum Rhein Main GmbH
Frankfurt am Main, Hesse, 60596, Germany
Universitatsklinikum Munster
Münster, North Rhine-Westphalia, 48149, Germany
Universitatsklinikum Leipzig
Leipzig, Saxony, 4103, Germany
Klinikum St. Georg Leipzig
Leipzig, Saxony, 4129, Germany
Universitaetsklinikum Schleswig-Holstein - Campus Luebeck
Lübeck, Schleswig-Holstein, 23538, Germany
Charite Campus Virchow-Klinikum-Augustenburger Platz 1
Berlin, 13353, Germany
Hadassah Medical Center- Ein Kerem - PPDS
Jerusalem, 9112001, Israel
Schneider Childrens Medical Center of Israel Petah Tikvah PIN
Petah Tikva, 4910000, Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906, Israel
Childrens University Hospital
Belgrade, 11000, Serbia
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
Belgrade, 11000, Serbia
Children and Youth Health Care Institute of Vojvodina
Novi Sad, 21000, Serbia
Royal Victoria Infirmary
Newcastle upon Tyne, Tyne and Wear, NE1 4LP, United Kingdom
The Royal London Hospital - PPDS
London, E1 1FR, United Kingdom
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 19, 2025
First Posted
November 26, 2025
Study Start
February 5, 2026
Primary Completion (Estimated)
June 15, 2026
Study Completion (Estimated)
June 15, 2026
Last Updated
May 5, 2026
Record last verified: 2026-04
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.
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.