The Impact of Charcot-Marie-Tooth Disease in the Real World
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 3,321
- 试验地点
- 1
- 主要终点
- EuroQoL 5-dimension 5-level (EQ-5D-5L)
研究概览
简要总结
An observational, non-interventional registry study to collect real-world data from people living with Charcot-Marie-Tooth disease (CMT) and its treatment, which will be available to researchers to further the knowledge of Charcot-Marie-Tooth disease and improve patient care.
详细描述
The registry uses Vitaccess' digital real-world evidence platform and has been developed in collaboration with CMT experts, Patient Advocacy Organizations (PAOs) and the biopharmaceutical company Pharnext.
Eligible participants install a study app on their smartphone. Researchers access aggregated, anonymised data via a cloud-based research portal.
The platform provides benefits to participants, which it is hoped will encourage persistence with data submission; these include options to download a copy of their symptom diary, a clinically-validated Knowledge feature, and access to high-level results from the study data.
The aggregated data are available in close to real time via "dashboards" and can be analysed according to a number of pre-set criteria (e.g. disease stage, age, geographic location).
State-of-the-art technologies and security policies are used in the platform to ensure industry-standard data storage and privacy for all users. Participants' personally identifiable information will remain confidential at all times, and researchers will not be able to identify individuals.
研究设计
- 研究类型
- Observational
- 观察模型
- Ecologic Or Community
- 时间视角
- Prospective
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
- •(*) Aged 18 years and over (*) Diagnosed with any stage and any subtype of Charcot-Marie-Tooth disease (*) Resident in France, Germany, Italy, Spain, the UK, or the USA (*) Have a National Health Service (NHS) or Community Health Index (CHI) number, or equivalent (*) Willing to use their own smartphone
排除标准
- •(*) No specific exclusion criteria.
结局指标
主要结局
EuroQoL 5-dimension 5-level (EQ-5D-5L)
时间窗: Throughout study completion, up to 62 months
The EQ-5D-5L comprises two parts - the EQ-5D-5L descriptive system and the EQ Visual Analogue Scale (EQ VAS). The descriptive system comprises 5 dimensions (mobility, self care, usual activities, pain/discomfort, and anxiety/depression), each with 5 levels (no problems, slight problems, moderate problems, severe problems, and extreme problems - i.e., higher scores represent worse health). The digits for 5 dimensions can be combined in a 5-digit number describing the respondent's health state. The EQ VAS records the respondent's self-rated health on vertical, visual analogue scale with endpoints labelled 'the best health you can imagine' and 'the worst health you can imagine'. Higher scores represent better self-perceived health.
次要结局
- Work Limitations Questionnaire (WLQ)(Throughout study completion, up to 62 months)
- Brief Fatigue Inventory (BFI)(Throughout study completion, up to 62 months)
- Falls Efficacy Scale - International (FES-I)(Throughout study completion, up to 62 months)
- Patient-Reported Outcomes Measurement Information System (PROMIS) Sleep Disturbance 8a(Throughout study completion, up to 62 months)
- Lower Extremity Function Scale (LEFS)(Throughout study completion, up to 62 months)
- Patient-Reported Outcomes Measurement Information System (PROMIS) Pain Intensity 3a and Interference 8a(Throughout study completion, up to 62 months)
- Bespoke questionnaire for this study(Throughout study completion, up to 62 months)
- QuickDash (Disabilities of the Arm, Shoulder and Hand)(Throughout study completion, up to 62 months)
研究者
Mark Larkin
Director
Vitaccess Ltd