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Real-World data to show if HAE drug works in kids

NCT ID NCT07251933

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 15, 2026 · Updated 2 times

Summary

This study tracks 40 children aged 2 to 12 with hereditary angioedema (HAE) who are taking lanadelumab to prevent sudden, painful swelling attacks. Researchers will look at medical records to see how long kids stay attack-free and how the drug performs in daily life. No new treatments are given; the goal is to understand real-world effectiveness and safety.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
lanadelumab (Takhzyro)
What this could lead to
If successful, this study could confirm that lanadelumab effectively prevents swelling attacks in children with HAE in everyday settings.
What could go wrong
This is an observational study using existing records, not a controlled trial, so results may be less reliable. It is also small (40 children) and focused on a rare condition.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

37 people

The number who actually took part.

Started

Feb 2026

Finished

Jul 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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

Ages

2 to 11 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • AP-HM- Hopital de La Timone

    Marseille, Bouches-du-Rhone, 13005, France

  • AP-HP - Hopital Armand Trousseau

    Paris, 75012, France

  • CHU de Grenoble Alpes - Hopital Couple-Enfant

    La Tronche, Grenoble, 38700, France

  • Centro de Alergia e Inmunologia Clinica Mar del Plata

    Mar del Plata, Buenos Aires, 7600, Argentina

  • Charite Campus Virchow-Klinikum-Augustenburger Platz 1

    Berlin, 13353, Germany

  • Children and Youth Health Care Institute of Vojvodina

    Novi Sad, 21000, Serbia

  • Childrens University Hospital

    Belgrade, 11000, Serbia

  • Hadassah Medical Center- Ein Kerem - PPDS

    Jerusalem, 9112001, Israel

  • Hamophilie Zentrum Rhein Main GmbH

    Frankfurt am Main, Hesse, 60596, Germany

  • Hospital Italiano de Buenos Aires

    Buenos Aires, Ciudad Autonoma de BuenosAires, C1199ABB, Argentina

  • Hospital de Ninos Sor Maria Ludovica

    La Plata, Buenos Aires, B1904CSI, Argentina

  • Hospital de Pediatria S.A.M.I.C.- Prof. Dr. Juan P. Garrahan

    Ciudad de Buenos Aires, Buenos Aires F.D., C1249ABN, Argentina

  • Klinikum St. Georg Leipzig

    Leipzig, Saxony, 4129, Germany

  • Mother and Child Health Care Institute of Serbia Dr Vukan Cupic

    Belgrade, 11000, Serbia

  • Royal Victoria Infirmary

    Newcastle upon Tyne, Tyne and Wear, NE1 4LP, United Kingdom

  • Sanatorio de la Canada-Cordoba

    Córdoba, X5000BSQ, Argentina

  • Schneider Childrens Medical Center of Israel Petah Tikvah PIN

    Petah Tikva, 4910000, Israel

  • Tel Aviv Sourasky Medical Center

    Tel Aviv, 6423906, Israel

  • The Royal London Hospital - PPDS

    London, E1 1FR, United Kingdom

  • Universitaetsklinikum Schleswig-Holstein - Campus Luebeck

    Lübeck, Schleswig-Holstein, 23538, Germany

  • Universitatsklinikum Frankfurt am Main

    Frankfurt am Main, Hesse, 60590, Germany

  • Universitatsklinikum Leipzig

    Leipzig, Saxony, 4103, Germany

  • Universitatsklinikum Munster

    Münster, North Rhine-Westphalia, 48149, Germany

  • Universitatsklinikum Ulm

    Ulm, Baden-Wurttemberg, 89081, Germany

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