The Impact of Charcot-Marie-Tooth Disease in the Real World

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

COMPLETED

Total Enrollment

3321 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-10-09

Study Completion Date

2023-12-31

Brief Summary

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An observational, non-interventional registry study to collect real-world data from people living with Charcot-Marie-Tooth disease (CMT) and its treatment, which will be available to researchers to further the knowledge of Charcot-Marie-Tooth disease and improve patient care.

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Detailed Description

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The registry uses Vitaccess' digital real-world evidence platform and has been developed in collaboration with CMT experts, Patient Advocacy Organizations (PAOs) and the biopharmaceutical company Pharnext.

Eligible participants install a study app on their smartphone. Researchers access aggregated, anonymised data via a cloud-based research portal.

The platform provides benefits to participants, which it is hoped will encourage persistence with data submission; these include options to download a copy of their symptom diary, a clinically-validated Knowledge feature, and access to high-level results from the study data.

The aggregated data are available in close to real time via "dashboards" and can be analysed according to a number of pre-set criteria (e.g. disease stage, age, geographic location).

State-of-the-art technologies and security policies are used in the platform to ensure industry-standard data storage and privacy for all users. Participants' personally identifiable information will remain confidential at all times, and researchers will not be able to identify individuals.

The study is ethics-approved in all scope countries and led by a Scientific Advisory Board comprising representatives from international PAOs, patients and key opinion leaders from each country, and study leads from both Pharnext Société Anonyme (SA) and Vitaccess Ltd.

Conditions

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Charcot-Marie-Tooth Disease

Study Design

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Observational Model Type

ECOLOGIC_OR_COMMUNITY

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

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Inclusion Criteria

(\*) Aged 18 years and over (\*) Diagnosed with any stage and any subtype of Charcot-Marie-Tooth disease (\*) Resident in France, Germany, Italy, Spain, the UK, or the USA (\*) Have a National Health Service (NHS) or Community Health Index (CHI) number, or equivalent (\*) Willing to use their own smartphone

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No