Clinical trial NCT06484478: A Real-world Wearables Study in Fabry Disease.
Official title: A Real-world Monitoring Project Exploring Day-to-day Physical Activity, Sleep, and Quality of Life in Individuals with Fabry Disease.
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Fabry Disease
Lead sponsor: Royal Free Hospital NHS Foundation Trust
Sponsor class: OTHER
Collaborators: name: Brunel University
First posted: 2024-07-03
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adults with Fabry disease who meet the eligibility criteria.

Brief 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.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of Fabry disease documented by genotyping and/or enzymatic analysis.
* Resident in the UK.
* Aged 18 years and over.
* Capable of providing informed consent.
* Physically able to stand and ambulate independently.

Exclusion Criteria:

* A known allergy to any materials in the wearable device.
* Not able to have or use a wearable.
* Taking part in an interventional study which would preclude real-world data collection.
* Unable to communicate in English to a sufficient level to permit engagement in the study.

Primary outcomes:
measure: Quality of life - via EuroQol five-dimension questionnaire (EQ-5D-5L).
timeFrame: Baseline
Secondary outcomes:
measure: Anxiety
timeFrame: Baseline
measure: Depression
timeFrame: Baseline
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: Royal Free London NHS Foundation Trust
Study officials:
References: