A Real-world Wearables Study in Fabry Disease.
- Conditions
- Fabry Disease
- Interventions
- Other: Wearable technology monitoring
- Registration Number
- NCT06484478
- Lead Sponsor
- Royal Free Hospital NHS Foundation Trust
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 100
- 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.
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Adults with Fabry disease Wearable technology monitoring -
- Primary Outcome Measures
Name Time Method Quality of life - via EuroQol five-dimension questionnaire (EQ-5D-5L). Baseline Quality of life measured using the self-report EuroQol five-dimension questionnaire (EQ-5D-5L). The questionnaire is comprised of five dimensions which are each divided into five levels of severity i.e. 1 = no problem, 2 = slight problems, 3 = moderate problems, 4 = severe problems, and 5 = unable to/extreme problems. A five-digit health state score is then found by combining the levels of severity for each dimension i.e. 11111 is the best health state and 55555 is the worst health state.
- Secondary Outcome Measures
Name Time Method Anxiety Baseline Anxiety measured using the self-report Hospital Anxiety and Depression Scale (HADS) questionnaire. HADS has 14 questions which measure anxiety (7 questions) and depression (7 questions). Each question is scored from 0 to 3 and total scores for anxiety (out of 21) and depression (out of 21) are calculated. Scoring is as follows: 0 - 7 = normal, 8 - 10 = borderline abnormal and 11 - 21 = abnormal.
Depression Baseline Depression measured using the self-report Hospital Anxiety and Depression Scale (HADS) questionnaire. HADS has 14 questions which measure anxiety (7 questions) and depression (7 questions). Each question is scored from 0 to 3 and total scores for anxiety (out of 21) and depression (out of 21) are calculated. Scoring is as follows: 0 - 7 = normal, 8 - 10 = borderline abnormal and 11 - 21 = abnormal.
Trial Locations
- Locations (1)
Royal Free London NHS Foundation Trust
🇬🇧London, United Kingdom