Assessing the multidimensional burden of facioscapulohumeral muscular dystrophy through patient-reported outcomes and experience.
Wenjing Ji, Yifei Zhao, Yuqiang Kang...
https://pubmed.ncbi.nlm.nih.gov/41739307Actively Recruiting
All Genders
ID06517498
Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
Led by Xi'an Jiaotong University · Updated on 2024-07-24
300
Participants Needed
1
Research Sites
17 weeks
Total Duration
On this page
Sponsors
X
Xi'an Jiaotong University
Lead Sponsor
C
Chinese Organization for Rare Disorders
Collaborating Sponsor
AI-Summary
What this Trial Is About
This observational study aims to understand the experiences of people with Facioscapulohumeral Muscular Dystrophy (FSHD) across all ages and genders. It focuses on learning about the patient journey, the burden of the disease, living situations, and quality of life. The study seeks to answer key questions about how FSHD affects patients and their daily lives. Participants will complete a detailed questionnaire about their health, symptoms, treatments, and how FSHD impacts them. They will also take part in in-depth interviews to share their personal experiences with the illness, including economic challenges and overall well-being. Medical experts on FSHD will be interviewed to provide insights into the disease and patient care. Throughout the study, researchers will collect data on quality of life, anxiety levels, economic burden, and disease burden over one year. This information will help identify areas where healthcare and support for people with FSHD can be improved. Participation involves questionnaires and interviews, with no medical interventions or treatments provided as part of the study.
CONDITIONS
Brief Title
Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
Who Can Participate
All Genders
Eligibility Criteria
You may qualify if you...
- Having been diagnosed as Facioscapulohumeral Muscular Dystrophy
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Diagnostic Evaluation
Duration - 1 day
Participants complete detailed questionnaires and undergo in-depth interviews to provide comprehensive data on their health status, disease burden, and quality of life related to Facioscapulohumeral Muscular Dystrophy.
1 visit (in-person or remote)
Long-term Monitoring
Duration - 1 year
Participants are followed over a 1-year period to assess ongoing disease burden, economic impact, anxiety levels, and quality of life through periodic assessments.
Periodic assessments during 1 year
Trial Site Locations
Total: 1 location
1
Health Science Center of Xi'an Jiaotong University
Xi'an, Shaanxi, China, 710049
Actively Recruiting
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Research Team
W
Wenjing Ji, PhD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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