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Completed

NCT Number: NCT07251933

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

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.

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Key information

Conditions

Hereditary Angioedema (HAE) Angioedema Angioedemas, Hereditary Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Complement Deficiency Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Immunologic Deficiency Syndromes Primary Immunodeficiency Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Age range

2 year–11 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital de Ninos Sor Maria Ludovica, La Plata, Buenos Aires, Argentina

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

No intervention

Other

This is a non-interventional study.

Primary outcomes

  1. 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).

Secondary outcomes

  1. HAE Attack Rate of Participants

    Time frame: Up to 13 months

  2. HAE Attack-free Rate of Participants Relative to Prior Treatment

    Time frame: Up to 13 months

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. Average Duration of Lanadelumab Treatment

    Time frame: Up to 13 months

    Average duration of lanadelumab treatment will be reported.

  8. 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.

  9. 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.

  10. 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.

  11. 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.

  12. Percentage of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Up to 13 months

  13. Percentage of Participants With Injection Site Reactions

    Time frame: Up to 13 months

    Percentage of participants with injection site reactions will be reported.

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

A Multi-national Real-world Outcomes and Treatment Patterns Study of Lanadelumab (Takhzyro) in Paediatric Patients With Hereditary Angioedema (TAHORA)

Important dates

Study start
2026
Primary completion
2026
Study completion
2026
First posted
Nov 26, 2025
Registry last updated
Jul 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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