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The United Kingdom Facioscapulohumeral Muscular Dystrophy Patient Registry

ConditionFacioscapulohumeral Muscular Dystrophy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorNewcastle University

About this trial

Facioscapulohumeral Dystrophy (FSHD) is the third most common form of neuromuscular dystrophy worldwide with an estimated prevalence of one in 20,000. FSHD is an autosomal dominant genetic disease and is estimated to affect up to 3,000 people in the UK.

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

Any confirmed NMD other than FSHD

Living outside of the UK

Trial population

Participants with FSHD volunteer to participate in this study. The registry is advertised through neuromuscular disease clinics, the registry website, patient organisations and conferences and meetings throughout the UK.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Patient Registry

    Other intervention

    Participants who have volunteered to participate will complete various questionnaires relating to their condition.

Treatment groups

1,018 Participants
are divided into 1 treatment group
Group A: Participants with FSHD1 intervention

Trial outcomes

Primary outcomes

1

Patient questionnaire

Patient reported FSHD clinical diagnosis, symptoms relating to muscle weakness, motor function, ventilation, retinal vascular disease, hearing loss, scapular fixation, family history and ethnicity.

Time frame
12 months
2

McGill Pain Questionnaire

Patient reported current pain.

Time frame
12 months
3

FSHD Pain Questionnaire

Patient reported experience of pain.

Time frame
12 months
4

The Short Form Health Survey (SF-36)

Patient reported quality of life.

Time frame
12 months

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Newcastle University

Lead sponsor

Source ClinicalTrials.gov
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