A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia

NCT07263685 | Not Yet Recruiting

• 1 other identifier: TAK-743-4037
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

Hereditary angioedema (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 (C1INH): HAE with normal C1INH levels (HAE-nC1INH) and HAE with limited or insufficient C1INH levels (HAE-C1INH); HAE-C1INH can be divided into Type 1, with low levels of C1INH, and Type 2, in which the protein is there, but does not work properly. This study will concentrate on people with HAE-C1INH Type 1 or 2 who have received Takhzyro® (lanadelumab) as prophylactic treatment for at least half a year (6 months). Prophylactic means that treatment is given to prevent the happening of HAE attacks. The main goal of the study is to see how well Takhzyro® works in everyday life to reduce the condition's activity after 6 months of treatment, or 12 months (if data is available). This will be measured by checking the change of the HAE activity from before treatment to after 6 months of treatment. The study design will permit a study follow-up of up to 12 months following the index event (i.e. date of first dose administration of Takhzyro®) unless the patient discontinues the index treatment, dies or is lost to follow-up within this timeframe. Chart abstractions will only occur once patients have at least 6 months' duration between the index event date and the date of chart abstraction initiation. Other goals are to find out how a person's quality of life changes after using Takhzyro® for 6 months, how often they had attacks before and after treatment and to learn which factors may have an impact on the treatment. 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)

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (1)

• Change in HAE- Activity Score (AS) From Pre to Post Index Period

  HAE-AS is a patient-reported outcome (PRO) instrument used to measure the activity of HAE. It comprises 12 items assessing factors like attack frequency, emergency visits, and impact on daily life over a recall period. A higher HAE-AS score indicates greater disease activity. A score of 13 or higher suggests severe HAE activity, while a score below 12 indicates mild or low activity.

  Up to 6 Months

Secondary Outcomes (3)

• Change in Quality of Life (QoL) Scores From Pre to Post Index Period

  Up to 6 months

• Change in HAE-C1INH Attack Frequency (number/month) From Pre to Post Index Period

  Up to 6 months

• Number of Participants Categorized by Demographic and Clinical Predictors of Treatment Response

  From Baseline up to end of study (up to 6 months)

Study Arms (1)

Participants with HAE-C1INH-Type 1 or HAE-C1INH-Type 2

All participants diagnosed with HAE-C1INH type 1 or type 2 will be included, and retrospective clinical data will be collected from patient medical records between 01 September 2025 and 28 February 2026. Participants who received Takhzyro® during the eligibility period beginning in 2021 will be observed across two periods: a pre-index period, defined as the time before the first dose of Takhzyro® (index date), used to establish baseline characteristics and disease activity, and a post-index period, defined as at least 6 months following the index date, used to assess treatment outcomes. The index date is defined as the date of the first Takhzyro® administration during the eligibility period.

Other: No intervention

Interventions

No intervention OTHER

This is a non-interventional study.

Participants with HAE-C1INH-Type 1 or HAE-C1INH-Type 2

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adolescents and adult participants with HAE-C1INH-Type 1 or HAE-C1INH-Type 2 treated with lanadelumab in routine clinical setting in the Kingdom of Saudi Arabia.

You may qualify if:

• Participant is diagnosed with HAE-C1INH-Type 1 or HAE-C1INH-Type 2 and initiated on long-term prophylaxis (LTP) with Takhzyro® (lanadelumab).
• Participant is aged greater than or equal to (\>=) 12 years at the time of Takhzyro® initiation.
• Participant has received at least 6 months of continuous treatment with Takhzyro® before data abstraction.

You may not qualify if:

• Participants who have normal C1INH function or HAE-nC1INH (formerly type III HAE).
• Participants who discontinued Takhzyro® before completing 6 months of treatment.
• Participants with insufficient or incomplete medical records which prevent the assessment of baseline HAE-AS at the time of Takhzyro® initiation, as well as 6 months HAE-AS after treatment initiation.
• Participants who are participating in an interventional clinical trial involving other HAE-C1INH treatments during the observation period.

Sponsors & Collaborators

• Takeda: lead

Study Sites (1)

King Faisal Specialist Hospital and Research Centre

Riyadh, 12713, Saudi Arabia

Location

Related Links

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

Conditions

Angioedemas, Hereditary

Condition Hierarchy (Ancestors)

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

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2025
• First Posted: December 4, 2025
• Study Start: April 30, 2026
• Primary Completion (Estimated): October 30, 2026
• Study Completion (Estimated): October 30, 2026
• Last Updated: May 4, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

SA (1)