The United Kingdom Facioscapulohumeral Muscular Dystrophy Patient Registry
NCT ID: NCT04001582
Last Updated: 2024-05-09
Study Results
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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RECRUITING
1018 participants
OBSERVATIONAL
2013-05-31
2040-01-31
Brief Summary
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The patient registry facilitates a questionnaire based research study to better characterise and understand the disease in the UK, and helps to identify potential participants eligible for clinical trials.
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Detailed Description
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The database is designed to be self reporting, however where specialised clinical or genetic information is required, the neuromuscular specialist in charge of the participants care can be invited to provide some additional information. The participant is able to select a health care provider from a pre-populated list at registration stage, if they wish to (optional feature). This information is included in the patient information and consent. Relevant R\&D approval has been recieved.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Participants with FSHD
Patients with a confirmed or pending diagnosis of FSHD, living in the UK are eligible to join the registry. Parents/guardians can register a child under 16 years old.
Patient Registry
Participants who have volunteered to participate will complete various questionnaires relating to their condition.
Interventions
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Patient Registry
Participants who have volunteered to participate will complete various questionnaires relating to their condition.
Eligibility Criteria
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Inclusion Criteria
Exclusion Criteria
* Living outside of the UK
ALL
No
Sponsors
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Newcastle University
OTHER
Responsible Party
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Principal Investigators
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Chiara Marini-Bettolo, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
John Walton Muscular Dystrophy Research Centre
Locations
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John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, , United Kingdom
Countries
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Central Contacts
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Facility Contacts
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References
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Moris G, Wood L, FernaNdez-Torron R, Gonzalez Coraspe JA, Turner C, Hilton-Jones D, Norwood F, Willis T, Parton M, Rogers M, Hammans S, Roberts M, Househam E, Williams M, Lochmuller H, Evangelista T. Chronic pain has a strong impact on quality of life in facioscapulohumeral muscular dystrophy. Muscle Nerve. 2018 Mar;57(3):380-387. doi: 10.1002/mus.25991. Epub 2017 Nov 7.
Ricci G, Cammish P, Siciliano G, Tupler R, Lochmuller H, Evangelista T. Phenotype may predict the clinical course of facioscapolohumeral muscular dystrophy. Muscle Nerve. 2019 Jun;59(6):711-713. doi: 10.1002/mus.26474. Epub 2019 Apr 4.
Evangelista T, Wood L, Fernandez-Torron R, Williams M, Smith D, Lunt P, Hudson J, Norwood F, Orrell R, Willis T, Hilton-Jones D, Rafferty K, Guglieri M, Lochmuller H. Design, set-up and utility of the UK facioscapulohumeral muscular dystrophy patient registry. J Neurol. 2016 Jul;263(7):1401-8. doi: 10.1007/s00415-016-8132-1. Epub 2016 May 9.
Related Links
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The UK FSHD Patient Registry website
Other Identifiers
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18/NE/0288
Identifier Type: -
Identifier Source: org_study_id
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