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

Официальное название

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)

Обзор

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.

Вмешательства

• Другое No intervention
  This is a non-interventional study.

Первичные конечные точки

• Change in HAE- Activity Score (AS) From Pre to Post Index Period [Срок оценки: Up to 6 Months]

Вторичные конечные точки (3)

• Change in Quality of Life (QoL) Scores From Pre to Post Index Period [Срок оценки: Up to 6 months]
• Change in HAE-C1INH Attack Frequency (number/month) From Pre to Post Index Period [Срок оценки: Up to 6 months]
• Number of Participants Categorized by Demographic and Clinical Predictors of Treatment Response [Срок оценки: From Baseline up to end of study (up to 6 months)]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Саудовская Аравия · 1 центр

• King Faisal Specialist Hospital and Research Centre — Riyadh

Идентификаторы

NCT: NCT07263685 · TAK-743-4037

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗