An observational study in Muscular Dystrophy, Facioscapulohumeral, sponsored by Xi'an Jiaotong University. Status unknown at 1 site in China. Per ClinicalTrials.gov, last updated 2024-07-24.
Sponsored by Xi'an Jiaotong University · Observational
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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:
Patients with Facioscapulohumeral Muscular Dystrophy will be asked to:
Medical experts on Facioscapulohumeral Muscular Dystrophy will be asked to be interviewed and answer questions about understanding on the disease and the patients.
Study Objective This observational study aims to comprehensively understand the patient journey, disease burden, living situation, and quality of life in individuals diagnosed with Facioscapulohumeral Muscular Dystrophy (FSHD). The study encompasses all sex/gender and age groups to provide a holistic view of the disease's impact.
Research Questions
The study focuses on answering the following key questions:
Methodology
To gather comprehensive data, participants will be engaged in two main activities:
1.Questionnaire Completion: Participants will fill out a detailed questionnaire designed to capture quantitative data on their health status, symptoms, treatments received, and the impact of FSHD on their daily lives. The questionnaire will also collect demographic information and other relevant background data.
2.In-Depth Interviews with patients with Facioscapulohumeral Muscular Dystrophy: They will undergo semi-structured interviews conducted by trained researchers. These interviews will delve into the personal experiences of living with FSHD, including:
3.In-Depth Interviews with medical experts on Facioscapulohumeral Muscular Dystrophy: They will undergo semi-structured interviews conducted by trained researchers. These interviews will delve into their understanding on the disease and the patients.
Expected Outcomes The study aims to generate a detailed understanding of the multifaceted impact of FSHD on patients' lives. By examining the patient journey, disease burden, and quality of life, the study seeks to identify gaps in current healthcare provisions and areas where additional support may be needed. This information will be valuable for healthcare providers, policymakers, and patient support organizations in improving care and support for individuals with FSHD.
79 studies on the registry are indexed under Muscular Dystrophy, Facioscapulohumeral; 26 are open to participants now.
This study's planned enrollment of 300 is above the median of 87 across 30 observational studies indexed under Muscular Dystrophy, Facioscapulohumeral.
Browse Muscular Dystrophy, Facioscapulohumeral studies →Xi'an Jiaotong University is the lead sponsor of 33 studies on the registry; 8 are open to participants now.
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The study will focus on individuals diagnosed as Facioscapulohumeral Muscular Dystrophy in all sex and age groups.
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Exclusion Criteria:
No intervention will be administered
Other: No Intervention
No Intervention will be conducted
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
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
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
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
Plan to share: No — No IPD will be shared
Started Mar 2024 (actual). Primary completion is estimated for Mar 2025, 12 months after the start.
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This study is status unknown, as verified in Jul 2024. You cannot join it, but the record below documents what was studied.
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Muscular Dystrophy, Facioscapulohumeral →
Xi'an Jiaotong University