Observational cohort study on Facioscapulohumeral Muscular Dystrophy Type 1
A Registered Observational Cohort Study of Facioscapulohumeral Muscular Dystrophy Type 1
This study is collecting information from people with Facioscapulohumeral Muscular Dystrophy Type 1 in China to better understand how the condition affects them and help doctors make better treatment decisions.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 1000 (estimated) |
| Sex | All |
| Sponsor | First Affiliated Hospital of Fujian Medical University Academic / other |
| Locations | 1 site (Fuzhou, Fujian) |
| Trial ID | NCT04369209 on ClinicalTrials.gov |
What this trial studies
This observational cohort study aims to collect data from genetically-confirmed FSHD1 patients across China, including those with and without symptoms. The study will gather retrospective and prospective data to enhance understanding of the prevalence, progression, and natural history of FSHD1. By analyzing this data, the study seeks to inform healthcare providers in making critical medical and financial decisions regarding the management of FSHD1.
Who should consider this trial
Good fit: Ideal candidates include male and female individuals of all ages with genetically-confirmed FSHD1.
Not a fit: Patients with other neuromuscular diseases or serious systemic illnesses may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights that improve the management and treatment of patients with FSHD1.
How similar studies have performed: While similar observational studies have been conducted, this specific cohort study is novel in its nationwide approach to FSHD1 in China.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Male or female subjects of all ages at baseline * Subjects, with or without symptoms, with FSHD1 genetic confirmation through PFGE-based Southern blotting * Unrelated healthy controls Exclusion Criteria: * Decline to participate * Other neuromuscular disease (such as Limb-girdle muscular dystrophy or Myotonic dystrophy) * Serious systemic illness (such as heart, liver, kidney disease or major mental illness)
Where this trial is running
Fuzhou, Fujian
- First Affiliated Hospital of Fujian Medical University — Fuzhou, Fujian, China (Recruiting)
Study contacts
- Study coordinator: Ning Wang
- Email: ningwang@fjmu.edu.cn
- Phone: 13805015340
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.