Observational Study to Describe Health-Related Quality of Life and Disease Burden Among Patients With Long QT Syndrome (LQTS) 2 and 3
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- Quality of Life (QoL)
研究概览
简要总结
This is a cross-sectional observational study designed to assess the disease burden and quality of life in individuals with LQTS 2 and 3. Each participant will answer each patient-reported outcome questionnaire only once over a 6-month period. The study will enroll up to 200 participants.
详细描述
The aim of the present cross-sectional observational study is to evaluate the health status and quality of life burden in patients with LQTS. The total study duration is anticipated to last for a period of up to 18 months, including up to 6 months of each individual's observation period. The observation period may be extended by up to 3 months (total 9 months). During the optional extension, data collection via myQTwave will be limited to emotional and physical symptoms survey. This prospective cohort study will collect PRO information in a cohort of patients with LQTS 2 or 3. All screened participants will be assigned a unique identifier. Eligible participants will download the myQTwave app on the Apple iPhone and Apple Watch delivered to them after enrollment.
Patient-reported outcomes will be captured via the Apple iPhone and daily health metrics (sleep, physical and Heart-related data) will be collected using the Apple Watch.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant is willing and provides written informed consent to participate in this study.
- •Male or female participant of at least 18 years of age, English-speaking.
- •Confirmed genetic diagnosis of LQT2 or 3, demonstrated by one of the following:
- •Genetic testing report (pathogenic or likely pathogenic (P/LP) mutation) in KCHN2 or SCN5a genes) or,
- •A signed physician's letter confirming genetic diagnosis of LQTS 2 or
- •Documented QTc ≥ 480 ms within the last year, demonstrated by one of the following:
- •12-lead electrocardiogram (ECG), or
- •A signed physician's letter confirming an ECG demonstrating QTc value ≥ 480 ms.
- •The participant is able to operate a smartphone and a companion watch. *The number of participants with QTc between 480 and 500 ms will be limited to 50.
排除标准
- •Current participation in another clinical trial involving a drug or device.
- •Participants unwilling to use an iPhone or Apple Watch for the duration of the study.
- •Known diagnosis of Brugada Syndrome
- •Participants unwilling to comply with outlined procedures.
结局指标
主要结局
Quality of Life (QoL)
时间窗: Month 1
Score from the Quality of Life in Life-Threatening Illness - Late Adolescents and Adults Scale (QOL-LAS)
Disease burden
时间窗: Month 2
Score from the Health Actions Evaluation (HAE-10)
Illness perception
时间窗: Month 3
Score from the Illness Intrusiveness Questionnaire (IIQ)
Psychosocial health, including distress, social and psychological well-being, and cardiac-specific measures,
时间窗: Month 3
Score from the Multidimensional Scale of Perceived Social Support (MSPSS)
次要结局
未报告次要终点