A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE) in the Kingdom of Saudi Arabia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: No intervention.
- Who it may be relevant to
- Registry conditions: Hereditary Angioedema (HAE). Basic parameters: from 12 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Saudi Arabia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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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)
Overview
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.
Interventions
- Other No intervention
This is a non-interventional study.
Primary outcome measures
- Change in HAE- Activity Score (AS) From Pre to Post Index Period [Time frame: Up to 6 Months]
Secondary outcome measures (3)
- 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)]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Saudi Arabia · 1 center
- King Faisal Specialist Hospital and Research Centre — Riyadh
Identifiers
NCT: NCT07263685 · TAK-743-4037