Hereditary Angioedema (HAE)NCT07251933
A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries
An observational study of Hereditary Angioedema (HAE), run by Takeda at 24 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 11 years
- Conditions the study is for
- Hereditary Angioedema (HAE)
- How many people
- 37 participants
- Where
- 24 sitesin 6 countries
- Study dates
- 5 Feb 2026to 5 Jul 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 11 years
- All sexes
- No healthy volunteers
- Child
Study population: Paediatric participants who have been diagnosed with HAE-C1INH in Germany, France, United Kingdom (UK), Israel, Argentina and Serbia.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
24 sites in 6 countries.
24 sites in 6 countries
Germany8 sites
Charite Campus Virchow-Klinikum-Augustenburger Platz 1
Berlin, 13353
Universitatsklinikum Frankfurt am Main
Frankfurt am Main, Hesse, 60590
Hamophilie Zentrum Rhein Main GmbH
Frankfurt am Main, Hesse, 60596
Universitatsklinikum Leipzig
Leipzig, Saxony, 4103
Klinikum St. Georg Leipzig
Leipzig, Saxony, 4129
Universitaetsklinikum Schleswig-Holstein - Campus Luebeck
Lübeck, Schleswig-Holstein, 23538
Universitatsklinikum Munster
Münster, North Rhine-Westphalia, 48149
Universitatsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081
Argentina5 sites
Hospital Italiano de Buenos Aires
Buenos Aires, Ciudad Autonoma de BuenosAires, C1199ABB
Hospital de Pediatria S.A.M.I.C.- Prof. Dr. Juan P. Garrahan
Ciudad de Buenos Aires, Buenos Aires F.D., C1249ABN
Sanatorio de la Canada-Cordoba
Córdoba, X5000BSQ
Hospital de Ninos Sor Maria Ludovica
La Plata, Buenos Aires, B1904CSI
Centro de Alergia e Inmunologia Clinica Mar del Plata
Mar del Plata, Buenos Aires, 7600
France3 sites
CHU de Grenoble Alpes - Hopital Couple-Enfant
La Tronche, Grenoble, 38700
AP-HM- Hopital de La Timone
Marseille, Bouches-du-Rhone, 13005
AP-HP - Hopital Armand Trousseau
Paris, 75012
Israel3 sites
Hadassah Medical Center- Ein Kerem - PPDS
Jerusalem, 9112001
Schneider Childrens Medical Center of Israel Petah Tikvah PIN
Petah Tikva, 4910000
Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
Serbia3 sites
Childrens University Hospital
Belgrade, 11000
Mother and Child Health Care Institute of Serbia Dr Vukan Cupic
Belgrade, 11000
Children and Youth Health Care Institute of Vojvodina
Novi Sad, 21000
United Kingdom2 sites
The Royal London Hospital - PPDS
London, E1 1FR
Royal Victoria Infirmary
Newcastle upon Tyne, Tyne and Wear, NE1 4LP
About this study
Official title: 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.
Read more
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.
- Conditions
- Hereditary Angioedema (HAE)
- Being tested
- No intervention
- Keywords
- Drug Therapy
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
This is a non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: Up to 13 months
Secondary outcomes (13)
HAE Attack Rate of Participants
Measured: Up to 13 months
HAE Attack-free Rate of Participants Relative to Prior Treatment
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to 13 months
Average Duration of Lanadelumab Treatment
Average duration of lanadelumab treatment will be reported.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to 13 months
Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Up to 13 months
Percentage of Participants With Injection Site Reactions
Percentage of participants with injection site reactions will be reported.
Measured: Up to 13 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2025
- Study start5 Feb 2026
- Primary completion5 Jul 2026
- Study completion5 Jul 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Angioedema (HAE)Studies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07251933
- Sponsor's protocol ID
- TAK-743-4038
- Status last verified
- Jul 2026
- First submitted
- 19 Nov 2025
- Last updated
- 14 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-national Real-world Outcomes and Treatment Patterns Study of Lanadelumab (Takhzyro) in Paediatric Patients With Hereditary Angioedema (TAHORA)
- ClinicalTrials.gov ID
- NCT07251933
- Acronym / sponsor ID
- TAK-743-4038
- Conditions
- Hereditary Angioedema (HAE)
- Keywords
- Drug Therapy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 5 Feb 2026 (actual)
- Primary completion
- 5 Jul 2026 (actual)
- Study completion
- 5 Jul 2026 (actual)
- First posted
- 26 Nov 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with HAE-C1INH | – | Other: No interventionPaediatric participants with HAE-C1INH who initiated long-term prophylaxis (LTP) treatment with lanadelumab within a routine clinical setting will be included. |
Who may qualify
- Ages
- 2 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Paediatric participants who have been diagnosed with HAE-C1INH in Germany, France, United Kingdom (UK), Israel, Argentina and Serbia.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionThis is a non-interventional study. | Other | Participants with HAE-C1INH |
Primary outcome
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).
13 secondary outcomes
HAE Attack Rate of Participants
Time frame: Up to 13 months
HAE Attack-free Rate of Participants Relative to Prior Treatment
Time frame: Up to 13 months
Association Between Covariates of Interest and Attack Free Rate
Time frame: 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 Location
Time frame: Up to 13 months
Number of Participants with Treatment History Before use of Lanadelumab
Time frame: Up to 13 months
Average Duration of Lanadelumab Treatment
Time frame: Up to 13 months
Number of Participants Characterized Based on Lanadelumab Treatment Patterns
Time frame: Up to 13 months
Number of Participants With Treatments Received Following Lanadelumab Discontinuation
Time frame: Up to 13 months
Number of Participants With Healthcare Resource Utilization (HRU) Outcomes
Time frame: Up to 13 months
Duration of Hospital Stay in Days Related to HAE-C1INH Hospitalizations
Time frame: Up to 13 months
Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to 13 months
Percentage of Participants With Injection Site Reactions
Time frame: Up to 13 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2026.
- Click here for more information about this trial in easy-to-understand language.Link provided by the sponsor.
- Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
24 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hereditary Angioedema (HAE), run by Takeda at 24 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in Germany, Argentina, France, Israel, Serbia and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants who are Free of HAE Attacks (time frame: Up to 13 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jul 2026.