A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries

April 29, 2026 updated by: Takeda

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

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

40

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Argentina
      • Córdoba, Argentina, X5000BSQ
        • Recruiting
        • Sanatorio de la Canada-Cordoba
        • Contact:
        • Principal Investigator:
          • Monica Marroco
    • Buenos Aires
      • La Plata, Buenos Aires, Argentina, B1904CSI
        • Not yet recruiting
        • Hospital de Niños Sor María Ludovica
        • Contact:
        • Principal Investigator:
          • Claudia Marcela Garcia
      • Mar del Plata, Buenos Aires, Argentina, 7600
        • Not yet recruiting
        • Centro de Alergia e Inmunologia Clinica Mar del Plata
        • Contact:
        • Principal Investigator:
          • Florencia Baillieau
    • Buenos Aires F.D.
      • Ciudad de Buenos Aires, Buenos Aires F.D., Argentina, C1249ABN
        • Not yet recruiting
        • Hospital de Pediatría S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
        • Contact:
        • Principal Investigator:
          • Matias Oleastro
    • Ciudad Autonoma de BuenosAires
      • Buenos Aires, Ciudad Autonoma de BuenosAires, Argentina, C1199ABB
        • Recruiting
        • Hospital Italiano de Buenos Aires
        • Principal Investigator:
          • Claudio Parisi
        • Contact:
  • France
      • Paris, France, 75012
        • Recruiting
        • AP-HP - Hôpital Armand Trousseau
        • Contact:
        • Principal Investigator:
          • Melissande Bourgoin-Heck
    • Bouches-du-Rhone
      • Marseille, Bouches-du-Rhone, France, 13005
        • Recruiting
        • AP-HM- Hôpital de La Timone
        • Contact:
        • Principal Investigator:
          • Stephane Gayet
    • Grenoble
      • La Tronche, Grenoble, France, 38700
        • Recruiting
        • CHU de Grenoble Alpes - Hopital Couple-Enfant
        • Contact:
        • Principal Investigator:
          • Anne Pagnier-Clemence
  • Germany
      • Berlin, Germany, 13353
        • Recruiting
        • Charité Campus Virchow-Klinikum-Augustenburger Platz 1
        • Contact:
        • Principal Investigator:
          • Renate Kruger
    • Baden-Wurttemberg
      • Ulm, Baden-Wurttemberg, Germany, 89081
        • Not yet recruiting
        • Universitätsklinikum Ulm
        • Contact:
        • Principal Investigator:
          • Robin Lochbaum
    • Hesse
      • Frankfurt am Main, Hesse, Germany, 60590
        • Not yet recruiting
        • Universitätsklinikum Frankfurt am Main
        • Principal Investigator:
          • Emel Aygoren-Pursun
        • Contact:
      • Frankfurt am Main, Hesse, Germany, 60596
        • Recruiting
        • Hamophilie Zentrum Rhein Main GmbH
        • Contact:
        • Principal Investigator:
          • Carmen Escuriola-Ettingshausen
    • North Rhine-Westphalia
      • Münster, North Rhine-Westphalia, Germany, 48149
        • Recruiting
        • Universitätsklinikum Münster
        • Contact:
        • Principal Investigator:
          • Mathias Sulk
    • Saxony
      • Leipzig, Saxony, Germany, 4103
      • Leipzig, Saxony, Germany, 4129
    • Schleswig-Holstein
      • Lübeck, Schleswig-Holstein, Germany, 23538
        • Recruiting
        • Universitaetsklinikum Schleswig-Holstein - Campus Luebeck
        • Principal Investigator:
          • Andreas Recke
        • Contact:
  • Israel
      • Jerusalem, Israel, 9112001
        • Recruiting
        • Hadassah Medical Center- Ein Kerem - PPDS
        • Contact:
        • Principal Investigator:
          • Aviv Talmon
      • Petah Tikva, Israel, 4910000
        • Recruiting
        • Schneider Childrens Medical Center of Israel Petah Tikvah PIN
        • Contact:
        • Principal Investigator:
          • Siril Yoffe
      • Tel Aviv, Israel, 6423906
        • Recruiting
        • Tel Aviv Sourasky Medical Center
        • Principal Investigator:
          • Shira Benor
        • Contact:
  • Serbia
      • Belgrade, Serbia, 11000
        • Recruiting
        • Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
        • Contact:
        • Principal Investigator:
          • Gordana Petrovic
      • Belgrade, Serbia, 11000
        • Not yet recruiting
        • Childrens University Hospital
        • Contact:
        • Principal Investigator:
          • Marina Atanaskovic-Markovic
      • Novi Sad, Serbia, 21000
        • Recruiting
        • Children and Youth Health Care Institute of Vojvodina
        • Contact:
        • Principal Investigator:
          • Andrea Duretic
  • United Kingdom
      • London, United Kingdom, E1 1FR
        • Recruiting
        • The Royal London Hospital - PPDS
        • Contact:
        • Principal Investigator:
          • Anthony Dorr
    • Tyne and Wear
      • Newcastle upon Tyne, Tyne and Wear, United Kingdom, NE1 4LP
        • Recruiting
        • Royal Victoria Infirmary
        • Contact:
        • Principal Investigator:
          • Stephen Owens

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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
Time Frame: Up to 13 months
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
Time Frame: Up to 13 months
Up to 13 months
HAE Attack-free Rate of Participants Relative to Prior Treatment
Time Frame: Up to 13 months
Up to 13 months
Association Between Covariates of Interest and Attack Free Rate
Time Frame: Up to 13 months
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
Time Frame: Up to 13 months
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
Time Frame: Up to 13 months
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
Time Frame: Up to 13 months
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
Time Frame: Up to 13 months
Average duration of lanadelumab treatment will be reported.
Up to 13 months
Number of Participants Characterized Based on Lanadelumab Treatment Patterns
Time Frame: Up to 13 months
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
Time Frame: Up to 13 months
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
Time Frame: Up to 13 months
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
Time Frame: Up to 13 months
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)
Time Frame: Up to 13 months
Up to 13 months
Percentage of Participants With Injection Site Reactions
Time Frame: Up to 13 months
Percentage of participants with injection site reactions will be reported.
Up to 13 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Takeda

Investigators

  • Study Director: Study Director, Takeda

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 5, 2026

Primary Completion (Estimated)

June 15, 2026

Study Completion (Estimated)

June 15, 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)

May 5, 2026

Last Update Submitted That Met QC Criteria

April 29, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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