Fabry disease Nutritional, Metabolic, Endocrine
Conditions
Interventions
Randomisation will be completed using an online randomisation tool, Research Randomizer (https://www.randomizer.org). An independent researcher will conduct the randomisation process. Participants wil
Sponsors
Brunel University of London
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Consultant confirmed diagnosis of Fabry disease 2. Aged =18 years old 3. Have a Functional Ambulation Category rating of =3 (3 = ambulation with supervision, 4 = independent ambulation on level surfaces, and 5 = independent ambulation in all situations) 4. Lives in the United Kingdom
Exclusion criteria
Exclusion criteria: 1. Unable to provide informed consent 2. Unable to communicate in English to a sufficient level to permit engagement in the study 3. Taking part in another interventional study 4. Any medical conditions which result in the clinician deeming the individual ineligible to participate (e.g. severe neuropathic pain, stage 4 heart failure or other condition resulting in breathlessness to a level that it could be unsafe to participate)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Feasibility, safety, and acceptability outcome measures calculated at the end of the study: 1. Participant eligibility will be calculated as: participants eligible / participants assessed for eligibility x 100 2. Recruitment rate will be calculated as: participants randomised / participants eligible x 100 3. Retention rate will be calculated as: participants who complete measures three months after intervention start / participants enrolled x 100 4. Completion rates for each outcome measure will be calculated as: complete data for the outcome measure / participants enrolled x 100 5. Trial safety will be assessed by calculating the frequency of pain crises, unscheduled hospital admissions, unscheduled GP appointments, any other adverse events and serious adverse events for the control and intervention groups 6. Acceptability of the intervention will be explored via focus groups with a subset of intervention participants | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary outcomes are preliminary estimates of intervention effects on: 1. Sedentary time measured using a physical activity monitor (activPAL4) over one full week 2. Physical activity measured using a physical activity monitor (activPAL4) over one full week 3. Anxiety measured using the Hospital Anxiety and Depression Scale (HADS) 4. Depression measured using the Hospital Anxiety and Depression Scale (HADS) 5. Quality of life measured using the EuroQol five-dimension questionnaire (EQ-5D-5L) 6. Goal attainment measured using Goal Attainment Scaling (GAS) Sedentary time, physical activity, anxiety, depression, and quality of life will all be measured at baseline and 3 months after intervention start. Goal attainment will be measured 3 months after intervention start. | — |
Countries
England, United Kingdom
Contacts
Public ContactSarah Gosling
Outcome results
None listed