King Faisal Specialist Hospital and Research Centre
Riyadh, 12713, Saudi Arabia
Location status: Recruiting
NCT Number: NCT07263685
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.
Interested in participating?
Request Info12 year and older
All sexes
Observational
Riyadh, 12713, Saudi Arabia
Location status: Recruiting
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Inclusion criteria
Exclusion criteria
This is a non-interventional study.
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.
Time frame: Up to 6 months
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.
Time frame: Up to 6 months
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.
Time frame: From Baseline up to end of study (up to 6 months)
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.
Contact information is provided by the study sponsor or research team.
Takeda
Industry
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)
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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