Fabry DiseaseNCT06257901
A Co-designed Physical Activity Intervention in Fabry Disease
An observational study of Fabry Disease, run by Brunel University at 1 site in United Kingdom.
Trial summary
Currently, treatments for Fabry disease are pharmacological and predominantly focus on the physical symptoms of the disease. In the general population and individuals with disabilities, increasing physical activity levels and reducing sedentary time can be an effective, non-pharmacological treatment to improve mental… Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Interviews/Focus groups/Participatory workshops
- How many people
- 41 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 1 Mar 2024to 30 Sep 2024
- 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
- Healthy volunteers accepted
- Adult, Older adult
Study population: Adults with Fabry disease, lay specialist stakeholders and specialist stakeholders who meet the eligibility criteria.
Who may be able to join
None listed.
Who cannot join
- Incapacity to provide written informed consent.
- Unable to communicate in English to a sufficient level to permit engagement in the study.
- Specialist stakeholders will be members of staff at NHS specialist centres for Fabry disease who are directly involved in the healthcare of adults with Fabry disease.
- Lay specialist stakeholders will be individuals who have been involved in supporting adults with Fabry disease, such as family, friends and members of staff at the Society for Mucopolysaccharide Diseases.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Co-design of a Physical Activity and Sedentary Behaviour Intervention for Adults with Fabry Disease
Currently, treatments for Fabry disease are pharmacological and predominantly focus on the physical symptoms of the disease. In the general population and individuals with disabilities, increasing physical activity levels and reducing sedentary time can be an effective, non-pharmacological treatment to improve mental health and quality of life. Such interventions have not yet been developed or evaluated in people with Fabry disease.
The aim of this study is to co-design a physical activity and sedentary behaviour intervention tailored to the needs of adults with Fabry disease. The study will seek to gain the expertise of adults with Fabry disease, specialist stakeholders (physicians, cardiologists and clinical nurse specialists) and lay specialist stakeholders (family and friends of adults with Fabry disease and members of staff and volunteers at the Society for Mucopolysaccharide Diseases). A range of views and experiences of physical activity and sedentary behaviour will be explored via focus groups (with individuals with Fabry disease and lay specialist stakeholders) and semi-structured interviews (with specialist stakeholders). The information gathered from the focus groups and interviews will then be utilised to inform participatory workshops (with individuals with Fabry disease) to test intervention concepts. Data from these activities will inform the design of a future intervention.
- Conditions
- Fabry Disease
- Being tested
- Interviews/Focus groups/Participatory workshops
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Interviews/Focus groups/Participatory workshopsOther
Qualitative study (no intervention delivered)
| Study group | Role | What they receive |
|---|---|---|
| Adults with Fabry disease | Focus groups and participatory workshops Receives: Other: Interviews/Focus groups/Participatory workshops | |
| Lay specialist stakeholders | Focus groups Receives: Other: Interviews/Focus groups/Participatory workshops | |
| Specialist stakeholders | Interviews Receives: Other: Interviews/Focus groups/Participatory workshops |
What is being measured
The primary outcome is the main question the study is designed to answer.
Focus groups (adults with Fabry disease)
Themes describing adults with Fabry disease's views and experiences of physical activity and sedentary behaviour- Emergence of themes describing participants' views and experiences from analysis of focus groups (using the Framework Method).
Measured: 9 months
Focus groups (lay specialist stakeholders)
Themes describing lay specialist stakeholders' views and experiences of physical activity and sedentary behaviour- Emergence of themes describing participants' views and experiences from analysis of focus groups (using the Framework Method).
Measured: 9 months
Semi-structured interviews (specialist stakeholders)
Themes describing specialist stakeholders' views and experiences of physical activity and sedentary behaviour- Emergence of themes describing participants' views and experiences from analysis of interviews (using the Framework Method).
Measured: 9 months
Participatory workshops (adults with Fabry disease)
Themes describing adults with Fabry disease's views and experiences of initial intervention concepts- Emergence of themes describing participants' views and experiences from analysis of participatory workshops (using the Framework Method).
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Feb 2024
- Study start1 Mar 2024
- Primary completion30 Sep 2024
- Study completion30 Sep 2024
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
- Other
- Time perspective
- Cross-sectional
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Brunel University
- Sponsor type
- University, hospital or other
- Collaborators
- Royal Free Hospital NHS Foundation Trust
- Responsible party
- Principal investigator, Dr Daniel Bailey, Brunel University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06257901
- Sponsor's protocol ID
- 45091-NHS-Oct/2023- 47368-1
- Status last verified
- Feb 2025
- First submitted
- 25 Jan 2024
- Last updated
- 13 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Co-design of a Physical Activity and Sedentary Behaviour Intervention for Adults with Fabry Disease
- ClinicalTrials.gov ID
- NCT06257901
- Acronym / sponsor ID
- 45091-NHS-Oct/2023- 47368-1
- Conditions
- Fabry Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Brunel UniversityUniversity, hospital or other
- Collaborators
- Royal Free Hospital NHS Foundation Trust
- Study start
- 1 Mar 2024 (actual)
- Primary completion
- 30 Sep 2024 (actual)
- Study completion
- 30 Sep 2024 (actual)
- First posted
- 14 Feb 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.
Currently, treatments for Fabry disease are pharmacological and predominantly focus on the physical symptoms of the disease. In the general population and individuals with disabilities, increasing physical activity levels and reducing sedentary time can be an effective, non-pharmacological treatment to improve mental health and quality of life. Such interventions have not yet been developed or evaluated in people with Fabry disease.
The aim of this study is to co-design a physical activity and sedentary behaviour intervention tailored to the needs of adults with Fabry disease. The study will seek to gain the expertise of adults with Fabry disease, specialist stakeholders (physicians, cardiologists and clinical nurse specialists) and lay specialist stakeholders (family and friends of adults with Fabry disease and members of staff and volunteers at the Society for Mucopolysaccharide Diseases). A range of views and experiences of physical activity and sedentary behaviour will be explored via focus groups (with individuals with Fabry disease and lay specialist stakeholders) and semi-structured interviews (with specialist stakeholders). The information gathered from the focus groups and interviews will then be utilised to inform participatory workshops (with individuals with Fabry disease) to test intervention concepts. Data from these activities will inform the design of a future intervention.
Study design
- Observational model
- Other
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Adults with Fabry disease | – | Other: Interviews/Focus groups/Participatory workshopsFocus groups and participatory workshops |
| Lay specialist stakeholders | – | Other: Interviews/Focus groups/Participatory workshopsFocus groups |
| Specialist stakeholders | – | Other: Interviews/Focus groups/Participatory workshopsInterviews |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Adults with Fabry disease, lay specialist stakeholders and specialist stakeholders who meet the eligibility criteria.
Key inclusion criteria (0 listed)
Key exclusion criteria (4 listed)
- Incapacity to provide written informed consent.
- Unable to communicate in English to a sufficient level to permit engagement in the study.
- Specialist stakeholders will be members of staff at NHS specialist centres for Fabry disease who are directly involved in the healthcare of adults with Fabry disease.
- Lay specialist stakeholders will be individuals who have been involved in supporting adults with Fabry disease, such as family, friends and members of staff at the Society for Mucopolysaccharide Diseases.
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 |
|---|---|---|
| Interviews/Focus groups/Participatory workshopsQualitative study (no intervention delivered) | Other | Adults with Fabry disease, Lay specialist stakeholders, Specialist stakeholders |
Primary outcome
Focus groups (adults with Fabry disease)
Time frame: 9 months
Themes describing adults with Fabry disease's views and experiences of physical activity and sedentary behaviour- Emergence of themes describing participants' views and experiences from analysis of focus groups (using the Framework Method).
Focus groups (lay specialist stakeholders)
Time frame: 9 months
Themes describing lay specialist stakeholders' views and experiences of physical activity and sedentary behaviour- Emergence of themes describing participants' views and experiences from analysis of focus groups (using the Framework Method).
Semi-structured interviews (specialist stakeholders)
Time frame: 9 months
Themes describing specialist stakeholders' views and experiences of physical activity and sedentary behaviour- Emergence of themes describing participants' views and experiences from analysis of interviews (using the Framework Method).
Participatory workshops (adults with Fabry disease)
Time frame: 9 months
Themes describing adults with Fabry disease's views and experiences of initial intervention concepts- Emergence of themes describing participants' views and experiences from analysis of participatory workshops (using the Framework Method).
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 13 Feb 2025.
- Individual participant dataSharing planned: No
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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 Brunel University 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 0 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 13 Feb 2025. 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?
Focus groups (adults with Fabry disease) (time frame: 9 months). The study lists 4 primary outcomes in all. 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 13 Feb 2025.