Fabry DiseaseNCT06484478
A Real-world Wearables Study in Fabry Disease.
An observational study of Fabry Disease, run by Royal Free Hospital NHS Foundation Trust at 1 site in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Wearable technology monitoring
- How many people
- 100 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 5 Nov 2024to 29 May 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Adults with Fabry disease who meet the eligibility criteria.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Real-world Monitoring Project Exploring Day-to-day Physical Activity, Sleep, and Quality of Life in Individuals With Fabry Disease.
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.
Read the 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
- Being tested
- Wearable technology monitoring
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Wearable technology monitoringOther
Observational study (no intervention delivered)
| Study group | Role | What they receive |
|---|---|---|
| Adults with Fabry disease | Not described Receives: Other: Wearable technology monitoring |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quality of life - via EuroQol five-dimension questionnaire (EQ-5D-5L).
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.
Measured: Baseline
Secondary outcomes (2)
Anxiety
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.
Measured: Baseline
Depression
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.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jul 2024
- Study start5 Nov 2024
- Primary completion29 May 2025
- Study completion29 May 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Royal Free Hospital NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- Brunel University
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06484478
- Sponsor's protocol ID
- 320134
- Status last verified
- Mar 2026
- First submitted
- 12 Jun 2024
- Last updated
- 16 Mar 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Real-world Monitoring Project Exploring Day-to-day Physical Activity, Sleep, and Quality of Life in Individuals With Fabry Disease.
- ClinicalTrials.gov ID
- NCT06484478
- Acronym / sponsor ID
- 320134
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Royal Free Hospital NHS Foundation TrustUniversity, hospital or other
- Collaborators
- Brunel University
- Study start
- 5 Nov 2024 (actual)
- Primary completion
- 29 May 2025 (actual)
- Study completion
- 29 May 2025 (actual)
- First posted
- 3 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Adults with Fabry disease | – | Other: Wearable technology monitoring |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Adults with Fabry disease who meet the eligibility criteria.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Wearable technology monitoringObservational study (no intervention delivered) | Other | Adults with Fabry disease |
Primary outcome
Quality of life - via EuroQol five-dimension questionnaire (EQ-5D-5L).
Time frame: 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.
2 secondary outcomes
Anxiety
Time frame: Baseline
Depression
Time frame: Baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Mar 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by Royal Free Hospital NHS Foundation Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quality of life - via EuroQol five-dimension questionnaire (EQ-5D-5L). (time frame: Baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Mar 2026.