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NCT Number: NCT06257901

A Co-designed Physical Activity Intervention in 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.

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Key information

Conditions

Fabry Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses Vascular Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Brunel University London

London, United Kingdom

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Individuals with Fabry disease will be eligible to participate in the study if they meet the following inclusion criteria:

  • Have been diagnosed with Fabry disease.
  • Aged ≥ 18 years old.
  • Able to independently ambulate with or without the use of a walking aid.
  • Lives in the United Kingdom.
  • Deemed eligible to participate following screening by a clinician (for participatory workshops).

The circumstances in which an individual may be deemed ineligible to participate in the participatory workshops based on the clinician's assessment are as follows:

  • Severe neuropathic pain.
  • Stage 4 heart failure or other condition resulting in breathlessness to a level they cannot participate.
  • Other clinical condition which dominates the clinical picture to the extent that it dominates the symptoms and biases the results.

Exclusion criteria

for all participants will be as follows:

  • 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.

Treatment and study plan

Interviews/Focus groups/Participatory workshops

Other

Qualitative study (no intervention delivered)

Primary outcomes

  1. 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).

  2. 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).

  3. 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).

  4. 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).

Sponsors and collaborators

Lead sponsor

Brunel University

Other

Collaborators

  • Royal Free Hospital NHS Foundation Trust

Registry information

Official study title

Co-design of a Physical Activity and Sedentary Behaviour Intervention for Adults with Fabry Disease

Important dates

Study start
2024
Primary completion
2024
Study completion
2024
First posted
Feb 14, 2024
Registry last updated
Feb 13, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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