Health TrialsbyHealthPanel

Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy

ConditionMuscular Dystrophy, Facioscapulohumeral
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorXi'an Jiaotong University

About this trial

The goal of this observational study is to learn the patient journey, disease burden, living situation, quality of life and etc. in patients with Facioscapulohumeral Muscular Dystrophy in all sex/gender and age groups. The main questions it aims to answer are:

1. What's the patient journey of patients with Facioscapulohumeral Muscular Dystrophy? 2. How does Facioscapulohumeral Muscular Dystrophy burden the patients? 3. How's the quality of life of patients with Facioscapulohumeral Muscular Dystrophy?

Patients with Facioscapulohumeral Muscular Dystrophy will be asked to:

1. fill a questionnaire. 2. be interviewed and answer questions about their illness experience, economic burden, quality of life and etc.

Medical experts on Facioscapulohumeral Muscular Dystrophy will be asked to be interviewed and answer questions about understanding on the disease and the patients.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Having been diagnosed as Facioscapulohumeral Muscular Dystrophy

Disqualifiers

No

Trial population

The study will focus on individuals diagnosed as Facioscapulohumeral Muscular Dystrophy in all sex and age groups.

Trial design

Design model

Ecologic or community

Time perspective

Retrospective

Treatments tested in this trial

  • No Intervention

    Other intervention

    No Intervention will be conducted

Treatment groups

300 Participants
are divided into 1 treatment group
Group A: Patients With Facioscapulohumeral Muscular Dystrophy1 intervention

Trial outcomes

Primary outcomes

1

EQ-5D

EQ-5D questionnaires will be used to measure the quality of life of the patients. The questionnaires consist of two main parts: Descriptive System and Visual Analog Scale. Descriptive System includes Mobility, Self-Care, Usual Activities, Pain/Discomfort and Anxiety/Depression, each with 5 levels for adults and 3 levels for children. Visual Analog Scalerequires patients to rate their overall health on a scale from 0 to 100, where 0 represents "the worst health you can imagine" and 100 represents "the best health you can imagine." In the end, an index value will be calculated that reflects the patient's overall health status.

Time frame
1 year
2

Anxiety Level

The Self-Rating Anxiety Scale will be used to measure the anxiety level of the patients. It consists of 20 items, each describing a common symptom of anxiety. Each item is rated on a 4-point Likert scale. The total score ranges from 20 to 80. In doing so, the anxiety level of the patients can be screened.

Time frame
1 year
3

Economic Burden

Questions will designed to explore the economic burden of the patients. Questions include direct and indirect medical costs over the past year, annual incomes, accumulated treatment costs and debts. Each questions include 7 levels. Besides, whether they have public health insurance and commercial insurance will be asked to discover how much the costs can be covered.

Time frame
1 year
4

Disease Burden

Questions will designed to explore the disease burden of the patients. Questions include the influence of the disease on respiratory system, dry eyes, whistle, deglutition, speaking and walking. Each question contains 5 levels.

Time frame
1 year

Secondary outcomes

Other outcomes

Sponsors and contacts

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

Xi'an Jiaotong University

Lead sponsor

Chinese Organization for Rare Disorders

Collaborator

First Affiliated Hospital of Fujian Medical University

Collaborator

Source ClinicalTrials.gov
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