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)

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.

Study Overview

• Status: Completed
• Conditions: Hereditary Angioedema (HAE)
• Intervention / Treatment: Other: No intervention

• Study Type: Observational
• Enrollment (Actual): 37

Contacts and Locations

Study Locations

• Argentina
  • Córdoba, Argentina, X5000BSQ
    • Sanatorio de la Canada-Cordoba
  • Buenos Aires
    • La Plata, Buenos Aires, Argentina, B1904CSI
      • Hospital de Ninos Sor Maria Ludovica
    • Mar del Plata, Buenos Aires, Argentina, 7600
      • Centro de Alergia e Inmunologia Clinica Mar del Plata
  • Buenos Aires F.D.
    • Ciudad de Buenos Aires, Buenos Aires F.D., Argentina, C1249ABN
      • Hospital de Pediatria S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
  • Ciudad Autonoma de BuenosAires
    • Buenos Aires, Ciudad Autonoma de BuenosAires, Argentina, C1199ABB
      • Hospital Italiano de Buenos Aires
• France
  • Paris, France, 75012
    • AP-HP - Hôpital Armand Trousseau
  • Bouches-du-Rhone
    • Marseille, Bouches-du-Rhone, France, 13005
      • AP-HM- Hôpital de La Timone
  • Grenoble
    • La Tronche, Grenoble, France, 38700
      • CHU de Grenoble Alpes - Hopital Couple-Enfant
• Germany
  • Berlin, Germany, 13353
    • Charité Campus Virchow-Klinikum-Augustenburger Platz 1
  • Baden-Wurttemberg
    • Ulm, Baden-Wurttemberg, Germany, 89081
      • Universitätsklinikum Ulm
  • Hesse
    • Frankfurt am Main, Hesse, Germany, 60590
      • Universitatsklinikum Frankfurt am Main
    • Frankfurt am Main, Hesse, Germany, 60596
      • Hamophilie Zentrum Rhein Main GmbH
  • North Rhine-Westphalia
    • Münster, North Rhine-Westphalia, Germany, 48149
      • Universitatsklinikum Munster
  • Saxony
    • Leipzig, Saxony, Germany, 4103
      • Universitätsklinikum Leipzig
    • Leipzig, Saxony, Germany, 4129
      • Klinikum St. Georg Leipzig
  • Schleswig-Holstein
    • Lübeck, Schleswig-Holstein, Germany, 23538
      • Universitaetsklinikum Schleswig-Holstein - Campus Luebeck
• Israel
  • Jerusalem, Israel, 9112001
    • Hadassah Medical Center- Ein Kerem - PPDS
  • Petah Tikva, Israel, 4910000
    • Schneider Childrens Medical Center of Israel Petah Tikvah PIN
  • Tel Aviv, Israel, 6423906
    • Tel Aviv Sourasky Medical Center
• Serbia
  • Belgrade, Serbia, 11000
    • Childrens University Hospital
  • Belgrade, Serbia, 11000
    • Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
  • Novi Sad, Serbia, 21000
    • Children and Youth Health Care Institute of Vojvodina
• United Kingdom
  • London, United Kingdom, E1 1FR
    • The Royal London Hospital - PPDS
  • Tyne and Wear
    • Newcastle upon Tyne, Tyne and Wear, United Kingdom, NE1 4LP
      • Royal Victoria Infirmary

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Paediatric participants who have been diagnosed with HAE-C1INH in Germany, France, United Kingdom (UK), Israel, Argentina and Serbia.

Description

Inclusion Criteria:

1. 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.
2. Participant has physician-confirmed diagnosis of HAE-C1INH.
3. Participant initiated LTP with lanadelumab during the eligibility period.
4. Signed consent/assent (where required by local regulations).
5. Participant's medical record contains documentation of HAE attacks in the pre-index period and after lanadelumab initiation.

Exclusion Criteria:

1. Participant was enrolled in a therapeutic investigational drug (lanadelumab or other drug) or device trial at index date.
2. Participant with no documented HAE attacks in the 12 months prior to index date.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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.	Other: No intervention; This is a non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Association between covariates of interest (e.g., age, sex, family history) and attack-free rate will be determined. Associations between attack free rate and covariates of interest will be modelled, using generalized linear models.	Up to 13 months
Number of Participants With HAE Attack Based on Attack Severity	Number of participants with HAE attack based on attack severity (mild, moderate, severe, unknown) will be reported.	Up to 13 months
Number of Participants With HAE Attack Based on Location	Number of participants with HAE attack based on location (e.g. extremities, abdominal, facial, laryngeal or genital) will be reported.	Up to 13 months
Number of Participants with Treatment History Before use of Lanadelumab	Number of participants with treatment history (type of treatment, duration of treatment, reason for start/ discontinuation/ switch) before use of lanadelumab will be reported.	Up to 13 months
Average Duration of Lanadelumab Treatment	Average duration of lanadelumab treatment will be reported.	Up to 13 months
Number of Participants Characterized Based on Lanadelumab Treatment Patterns	Number of participants based on lanadelumab treatment patterns (e.g. setting of administration, interval of administration changes/dose modifications, reasons for initiation/discontinuation and modifications) will be reported.	Up to 13 months
Number of Participants With Treatments Received Following Lanadelumab Discontinuation	Number of participants with treatments (e.g. type of treatment, reason for selection) received following lanadelumab discontinuation will be evaluated.	Up to 13 months
Number of Participants With Healthcare Resource Utilization (HRU) Outcomes	HRU outcomes will include number of healthcare professional (HCP) visits, number of emergency department (ED) visits, number of inpatient hospitalizations, number of participants with admission to and days spent in intensive care unit related to HAE-C1INH, and number of school days missed will be reported.	Up to 13 months
Duration of Hospital Stay in Days Related to HAE-C1INH Hospitalizations	Length of hospital stay in days related to HAE-C1INH hospitalizations will be reported.	Up to 13 months
Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)		Up to 13 months
Percentage of Participants With Injection Site Reactions	Percentage of participants with injection site reactions will be reported.	Up to 13 months

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

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• Click here for more information about this trial in easy-to-understand language.

Study record dates

Study Major Dates

• Study Start (Actual): February 5, 2026
• Primary Completion (Actual): July 5, 2026
• Study Completion (Actual): July 5, 2026

Study Registration Dates

• First Submitted: November 19, 2025
• First Submitted That Met QC Criteria: November 19, 2025
• First Posted (Actual): November 26, 2025

Study Record Updates

• Last Update Posted (Actual): July 14, 2026
• Last Update Submitted That Met QC Criteria: July 13, 2026
• Last Verified: July 1, 2026

More Information

Terms related to this study

• Keywords: Drug Therapy
• Additional Relevant MeSH Terms: Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Immune System Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immunologic Deficiency Syndromes; Skin Diseases; Urticaria; Skin Diseases, Vascular; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Angioedema; Angioedemas, Hereditary
• Other Study ID Numbers: TAK-743-4038

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No