A Real-world Wearables Study in Fabry Disease.

NCT06484478 | Completed

• 1 other identifier: 320134
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

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.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline

Secondary Outcomes (2)

• Anxiety

  Baseline

• Depression

  Baseline

Study Arms (1)

Adults with Fabry disease

Other: Wearable technology monitoring

Interventions

Wearable technology monitoring OTHER

Observational study (no intervention delivered)

Adults with Fabry disease

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adults with Fabry disease who meet the eligibility criteria.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Royal Free Hospital NHS Foundation Trust: lead
• Brunel University: collaborator

Study Sites (1)

Royal Free London NHS Foundation Trust

London, United Kingdom

Location

MeSH Terms

Conditions

Fabry Disease

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 12, 2024
• First Posted: July 3, 2024
• Study Start: November 5, 2024
• Primary Completion: May 29, 2025
• Study Completion: May 29, 2025
• Last Updated: March 16, 2026

Record last verified: 2026-03

Locations

GB (1)