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

NCT ID: NCT07263685

Last Updated: 2025-12-31

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Total Enrollment: 50 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2026-01-15
• Study Completion Date: 2026-05-31

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

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

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.

No intervention

Intervention Type: OTHER

This is a non-interventional study.

Interventions

No intervention

This is a non-interventional study.

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

1. Participant is diagnosed with HAE-C1INH-Type 1 or HAE-C1INH-Type 2 and initiated on long-term prophylaxis (LTP) with Takhzyro® (lanadelumab).

2. Participant is aged greater than or equal to (>=) 12 years at the time of Takhzyro® initiation.

3. Participant has received at least 6 months of continuous treatment with Takhzyro® before data abstraction.

Exclusion Criteria

1. Participants who have normal C1INH function or HAE-nC1INH (formerly type III HAE).

2. Participants who discontinued Takhzyro® before completing 6 months of treatment.

3. 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.

4. Participants who are participating in an interventional clinical trial involving other HAE-C1INH treatments during the observation period.

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Takeda

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

King Faisal Specialist Hospital and Research Centre

Riyadh, , Saudi Arabia

Countries

Saudi Arabia

Central Contacts

Takeda Contact

Role: CONTACT

Phone: +1-877-825-3327

Email: medinfoUS@takeda.com

Facility Contacts

Site Contact

Role: primary

Related Links

https://clinicaltrials.takeda.com/study-detail/ba5551a2b34748ad??page=1&idFilter=TAK-743-4037

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

Other Identifiers

TAK-743-4037

Identifier Type: -

Identifier Source: org_study_id