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A Real-world Wearables Study in Fabry Disease.

NCT ID: NCT06484478

Last Updated: 2024-12-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

100 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-11-05

Study Completion Date

2025-12-31

Brief Summary

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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

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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

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Fabry Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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Adults with Fabry disease

Wearable technology monitoring

Intervention Type OTHER

Observational study (no intervention delivered)

Interventions

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Wearable technology monitoring

Observational study (no intervention delivered)

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* 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.

Exclusion Criteria

* 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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Brunel University

OTHER

Sponsor Role collaborator

Royal Free Hospital NHS Foundation Trust

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Royal Free London NHS Foundation Trust

London, , United Kingdom

Site Status RECRUITING

Countries

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United Kingdom

Central Contacts

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Derralynn Hughes, Professor

Role: CONTACT

Phone: 020 7794 0500

Email: [email protected]

Sarah Gosling

Role: CONTACT

Email: [email protected]

Facility Contacts

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Derralynn Hughes, Professor

Role: primary

Other Identifiers

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320134

Identifier Type: -

Identifier Source: org_study_id