NCT06484478

A Real-world Wearables Study in Fabry Disease.

A Real-world Monitoring Project Exploring Day-to-day Physical Activity, Sleep, and Quality of Life in Individuals With Fabry Disease.

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 100 (actual)
• Sponsor: Royal Free Hospital NHS Foundation Trust · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Despite improvements in Fabry disease care in recent years, many patients still have poor outcomes. In addition to the many physical symptoms that patients can experience, the disease also places individuals at an increased risk of mental health conditions, such as anxiety and depression. Low physical activity levels and increased sedentary time are detrimentally associated with anxiety, depression and quality of life in the general population. Currently, no such research has been conducted in individuals with Fabry disease. Data will consist of: (1) information from hospital medical records, (2) patient reported outcomes collected via questionnaires, and (3) measurements from a clinical-grade wearable device. Individuals with Fabry disease aged 18 years or above will be considered for eligibility screening.

Detailed description

Trial assessments: Baseline questionnaires Baseline questionnaires will include the Hospital Anxiety and Depression Scale (HADS) and the EuroQol 5-dimension questionnaire (EQ-5D-5L). This will be administered via Qualtrics. Thigh-worn device (activPAL) Participants will attach the activPAL device to their anterior thigh for 8 days and continue their usual daily behaviour throughout the week. This will measure sitting, standing, stepping and sleep time. This device is accompanied by a hard copy daily diary, where participants will be asked to input the time they wake up, they get out of bed, they get into bed, and when they go to sleep. Clinical data Data from medical records will include disease phenotype and clinical severity including mutation type, lyso-Gb3, left ventricular mass index and diastolic dysfunction. Such data will be sourced from electronic patient records. Mainz Severity Score Index, and its age-adjusted score will be calculated from data on electronic patient records. These will be collected retrospectively from the previous 12 months upon enrolment.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
OTHER	Wearable technology monitoring	Observational study (no intervention delivered)

Timeline

• Start date: 2024-11-05
• Primary completion: 2025-05-29
• Completion: 2025-05-29
• First posted: 2024-07-03
• Last updated: 2026-03-16

Locations

1 site across 1 country: United Kingdom