Hereditary Angioedema (HAE)NCT07263685
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia
An observational study of Hereditary Angioedema (HAE), run by Takeda at 1 site in Saudi Arabia.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE)
- How many people
- 50 participants
- Where
- 1 siteSaudi Arabia
- Study dates
- 30 Apr 2026to 30 Oct 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Saudi Arabia1 site, 1 recruiting
- Recruiting
King Faisal Specialist Hospital and Research Centre
Riyadh, 12713
Site Contact, 00966 11 464 7272, rkarnaout@icloud.com
Rand Khaled Arnaout
About this study
Official title: Retrospective Chart Review Study Evaluating Clinical Effectiveness and Impact on Quality of Life Among Patients Who Initiated Long-term Prophylaxis With Takhzyro® in a Real-World Setting in the Kingdom of Saudi Arabia - the REFLEQT-KSA Study (Retrospective Evaluation Focusing on Lanadelumab's Effectiveness and Impact on Quality of Life in The Kingdom of Saudi Arabia)
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.
Read more
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)
- 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-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. Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 6 Months
Secondary outcomes (3)
Change in Quality of Life (QoL) Scores From Pre to Post Index Period
A study-specific quality-of-life (QoL) score will be developed. This custom score will be derived from select parameters conceptually aligned with the domains of the validated Angioedema (AE)-QoL (functioning, fatigue/mood, fear/shame, and nutrition), as captured in the available participant records. The goal is to approximate core aspects of health-related quality of life relevant to participants with recurrent angioedema, while ensuring feasibility and compliance within the constraints of retrospective data collection.
Measured: Up to 6 months
Change in HAE-C1INH Attack Frequency (number/month) From Pre to Post Index Period
HAE attack is defined as a discrete episode during which the participant progresses from no angioedema to symptoms of angioedema. A "change in HAE-C1INH attack frequency" refers to the reduction in the number of HAE attacks (number per month) after the implementation of treatments.
Measured: Up to 6 months
Number of Participants Categorized by Demographic and Clinical Predictors of Treatment Response
Number of participants will be reported by baseline demographic (age, sex) and clinical characteristics (disease duration, baseline attack frequency). The relationship between these characteristics and change from baseline in HAE-AS score and attack frequency will be further explored using regression analysis, provided sufficient sample size and model stability.
Measured: From Baseline up to end of study (up to 6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2025
- Study start30 Apr 2026
- Primary completion30 Oct 2026 (estimated)
- Study completion30 Oct 2026 (estimated)
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
- 50 participants (estimated)
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
- NCT07263685
- Sponsor's protocol ID
- TAK-743-4037
- Status last verified
- May 2026
- First submitted
- 24 Nov 2025
- Last updated
- 11 May 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 11 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Chart Review Study Evaluating Clinical Effectiveness and Impact on Quality of Life Among Patients Who Initiated Long-term Prophylaxis With Takhzyro® in a Real-World Setting in the Kingdom of Saudi Arabia - the REFLEQT-KSA Study (Retrospective Evaluation Focusing on Lanadelumab's Effectiveness and Impact on Quality of Life in The Kingdom of Saudi Arabia)
- ClinicalTrials.gov ID
- NCT07263685
- Acronym / sponsor ID
- TAK-743-4037
- Conditions
- Hereditary Angioedema (HAE)
- Keywords
- Drug Therapy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 30 Apr 2026 (actual)
- Primary completion
- 30 Oct 2026 (estimated)
- Study completion
- 30 Oct 2026 (estimated)
- First posted
- 4 Dec 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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Participants with HAE-C1INH-Type 1 or HAE-C1INH-Type 2 | – | Other: No interventionAll 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. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
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-Type 1 or HAE-C1INH-Type 2 |
Primary outcome
Change in HAE- Activity Score (AS) From Pre to Post Index Period
Time frame: Up to 6 Months
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.
3 secondary outcomes
Change in Quality of Life (QoL) Scores From Pre to Post Index Period
Time frame: Up to 6 months
Change in HAE-C1INH Attack Frequency (number/month) From Pre to Post Index Period
Time frame: Up to 6 months
Number of Participants Categorized by Demographic and Clinical Predictors of Treatment Response
Time frame: From Baseline up to end of study (up to 6 months)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 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.
Study Director study director
Takeda
1 site in 1 country: 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 1 site in Saudi Arabia.
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 3 inclusion and 4 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: looking for participants now, as of the sponsor's last update on 11 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Saudi Arabia. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Takeda Contact (+1-877-825-3327, medinfoUS@takeda.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in HAE- Activity Score (AS) From Pre to Post Index Period (time frame: Up to 6 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 11 May 2026.