Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06517498
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-07-09
Brief Title:
Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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 sexgender and age groups The main questions it aims to answer are
1 Whats the patient journey of patients with Facioscapulohumeral Muscular Dystrophy
2 How does Facioscapulohumeral Muscular Dystrophy burden the patients
3 Hows 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
1 Whats the patient journey of patients with Facioscapulohumeral Muscular Dystrophy
2 How does Facioscapulohumeral Muscular Dystrophy burden the patients
3 Hows 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
Detailed Description:
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 sexgender and age groups to provide a holistic view of the diseases impact
Research Questions
The study focuses on answering the following key questions
1 What is the patient journey of individuals with Facioscapulohumeral Muscular Dystrophy
2 How does Facioscapulohumeral Muscular Dystrophy burden the patients
3 What is the quality of life of patients with Facioscapulohumeral Muscular Dystrophy
Methodology
To gather comprehensive data participants will be engaged in two main activities
1Questionnaire 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
2In-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
1 Illness Experience Participants will describe their journey from initial symptoms to diagnosis and ongoing management of the disease
2 Economic Burden Discussions will cover the financial impact of FSHD including costs related to medical care treatments and any income loss due to the inability to work
3 Quality of Life Interviews will explore the effects of FSHD on various life domains such as physical health mental well-being social relationships and overall life satisfaction
3In-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
Research Questions
The study focuses on answering the following key questions
1 What is the patient journey of individuals with Facioscapulohumeral Muscular Dystrophy
2 How does Facioscapulohumeral Muscular Dystrophy burden the patients
3 What is the quality of life of patients with Facioscapulohumeral Muscular Dystrophy
Methodology
To gather comprehensive data participants will be engaged in two main activities
1Questionnaire 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
2In-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
1 Illness Experience Participants will describe their journey from initial symptoms to diagnosis and ongoing management of the disease
2 Economic Burden Discussions will cover the financial impact of FSHD including costs related to medical care treatments and any income loss due to the inability to work
3 Quality of Life Interviews will explore the effects of FSHD on various life domains such as physical health mental well-being social relationships and overall life satisfaction
3In-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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None